Gaucher Community News - National Gaucher Foundation

Who’s Who On NGF’s MedicalAdvisory BoardSpotlighting Dr. Roscoe Brady is part of an ongoingtribute we are paying to the NationalGaucher Foundation’s Medical Advisory Board.Page 2Roscoe O. Brady, M.D., Ph.D (h.c.)Scientist EmeritusNational Institutes of HealthBethesda, MDTribute to Dr. Roscoe BradyDr. Roscoe Brady obtained his M.D. degree fromHarvard Medical School. He interned at theHospital of the University of Pennsylvania and wasa post-doctoral fellow in the Department ofPhysiological Chemistry at the University ofPennsylvania School of Medicine and a fellow inclinical medicine in the Department of Medicine.Following two and one-half years on active duty inthe U.S. Naval Medical Corps, he joined theNational Institutes of Health where he was Chiefof the Developmental and Metabolic NeurologyBranch in the National Institute of NeurologicalDisorders and Stroke from 1972 to 2006. He isnow a scientist emeritus at the National Institutesof Health.Dr. Brady and his colleagues identified theenzymatic defects in Gaucher disease, Niemann-Pick disease, Fabry disease and the specificmetabolic abnormality in Tay-Sachs disease. Heand his associates developed diagnostic, carrierdetection and prenatal tests for the first three ofthese conditions.He and his coworkers developed effective enzymereplacement therapy for patients with Gaucher diseaseand Fabry disease. He is currently exploringmolecular chaperone therapy in Fabry disease andgene therapy in animal models of Fabry disease andGaucher disease.Dr. Brady received the Gairdner FoundationInternational Award in 1973; the American Academyof Neurology Cotzias Award in 1980; the PassanoFoundation Award and the Lasker FoundationClinical Medical Research Award in 1982; the SachsAward from the Child Neurology Society in 1990; theKovalenko Medal from the National Academy ofSciences USA in 1991 and the Alpert FoundationPrize from Harvard Medical School in 1992. He is amember of the National Academy of Sciences, USAand a member of the Institute of Medicine of theNational Academy of Sciences. He received anhonorary Ph.D. degree from the University ofUppsala, Sweden in 2005.Dr. R.O. Brady: Thesis Advisor,Mentor, and ColleagueIn 1995, I came to Dr. Brady¹s laboratory at the NationalInstitutes of Health in Bethesda, Maryland to do the practicalresearch part of my doctoral research with him. As a Germandoctoral student, I needed a German and an American thesisadvisor in order to obtain my degree at the University of Bonn.Professor Dr. Sandhoff, my German advisor, directs importantbasic research on lipid storage disorders in his laboratory. I wasthrilled when Dr. Brady agreed to be my co-thesis advisor,allowing me to advance my knowledge in his laboratory. I hadpreviously heard about Dr. Brady's groundbreaking research onlipid storage disorders, especially Gaucher disease.We worked on three relevant research questions concerningGaucher disease that became my thesis projects. Dr. Brady wasparticularly interested in two questions: first, whether the buildupof the toxic molecule glucopsychosine is responsible for theneuronal loss in the brains of type 2 or 3 Gaucher disease patients,and second, whether neurons would pick up and internalizeglucocerebrosidase so that infusion of the therapeutic enzymedirectly into the brain could prevent neuronal loss in thesepatients. The third research problem, which came from Dr.Sandhoff, was to provide some insight regarding the correlationof residual glucocerebrosidase enzyme activity and storage ofglucocerebroside, the accumulating lipid in patients withGaucher disease.

Chris Kaneski, and Jane Quirk Dr. Brady¹s assistants, trained me in the basiclaboratory assays and techniques I needed for my projects. Dr. Brady met withme every other week to discuss my progress, give me useful advice for the nextsteps, and showed me which scientific papers I could read to get a deeperunderstanding of the field. His immense knowledge always impressed me. Ihad the feeling that he liked very much to supervise my projects: he wasalways there for me if I had a question or needed help. Almost every day hecame by my workplace and asked me if I needed anything and if the projectwas going well. For me as a doctoral student it was just the right balancebetween supervision and responsibility for my own project. Dr. Brady wasalways more a guide then a pedagogue, so that I was able to learn to workindependently and enthusiastically since he made the questions we wanted toanswer so interesting.Another unique opportunity, I could only have had working with Dr. Brady,was to see the patients. In Dr. Sandhoff¹s laboratory we got diseased cells frompatients for research, but we never saw the people. Dr. Brady invited me everyweek to come with him on clinical rounds so that I could see how a patient withTay-Sachs, Niemann Pick C, Fabry or Gaucher disease type 1, 2 or 3 looks andwhat problems these people had to face. The experience and the knowledgethat I gained during these rounds were very important to me and changed mywhole attitude. I became aware of the importance of that little piece I could dowith my work to help change the lives of these people. Finally the samples onmy bench were not just a sample from some interesting disease but frompeople who where hoping that some day a cure could be found.In October 1999 Dr. Brady came to the University of Bonn were I defended mythesis titled, “Model Systems of Gaucher Disease”. Afterwards we publishedthree papers from our work:Schueler UH, Kolter T, Kaneski CR, Blusztajn JK, Herkenham M, Sandhoff K,Brady RO.Toxicity of glucosylsphingosine (glucopsychosine) to cultured neuronalcells: a model system for assessing neuronal damage in Gaucher diseasetype 2 and 3. Neurobiol Dis. 2003 Dec;14(3):595-601.Schueler U, Kaneski C, Murray G, Sandhoff K, Brady RO.Uptake of mannose-terminal glucocerebrosidase in cultured humancholinergic and dopaminergic neuron cell lines. Neurochem Res. 2002Apr;27(4):325-30.Schueler UH, Kolter T, Kaneski CR, Zirzow GC, Sandhoff K, Brady RO.Correlation between enzyme activity and substrate storage in a cell culturemodel system for Gaucher disease. J Inherit Metab Dis. 2004;27(5):649-58.Today I am working again at NIH but this time in NIDDK with Dr. Blanchette-Mackie on Niemann Pick C disease. I still enjoy dropping by Dr. Brady¹soffice from time to time to talk about my research and how life is treating usthese days.Dr. Ulrike H. SchuelerNational Institutes of HealthA Tribute From Cynthia FrankI like to say that Dr. Brady is the reason I'malive. I can't say that for sure, but he is thereason, enzyme replacement therapy aside, Inow lead a healthy and happy life.I first met Dr. Brady at 14 when I wasreferred to the National Institutes of Healthin 1977, a year after I was diagnosed withGaucher. I was a scared, rebellious andsomewhat angry teen, determined to doanything the doctors told me not to do.Contradictory to what my parents believed, Isaw no need traveling afar to spend time in ahospital that could do nothing for me exceptgauge how much worse I'd gotten from yearto year. Boy, was I wrong.For many years I visited Dr. Brady at theNIH once or twice a year. I got to know thestaff, I learned how to get around the hospitaland the Washington DC area on my own, andmost of all I became friends with Dr. Brady.He became a pillar of hope for my familyand me. His advice and guidance over theyears has lead me to make the right choices,and he has helped me to take care of mydisease the best I can. Through inviting meto participate in the enzyme replacementtherapy protocol at the NIH before treatmentwas approved by the FDA, he introduced meto others in the Gaucher community so that Ino longer felt alone with the disease. Andthrough encouragement he helped me to findmy path in life working within the Gauchercommunity myself with the NationalGaucher Foundation.In between NIH visits, I've seen many, manydoctors over the years, and I've been treatedlike a patient by most of them. Not so withDr. Brady. With Dr. Brady I found that I wasnot just a patient or a statistic, I was a personwith a disease whom he wanted to help.Never, in the past 30 years I've known him,has one email or one question goneunanswered or one phone call goneunreturned. His genuine caring and concernhas been the constant throughout the years.By being Dr. Brady's patient I've beenblessed with the friendship of a brilliant andmagnificent person who has given me hopeand taught me to have hope.Thank you Dr. Brady!Cynthia J. FrankNational Gaucher FoundationPage 3

HAPPY HOLIDAYSMERRY CHRISTMAS, HAPPY CHANUKAH,HAPPY NEW YEAR!By Rhonda Buyers, Executive DirectorHow can it be possible that 2007 is just around thecorner? I feel as though 2006 should only beabout half over! Each year seems to be busier andbusier, but I must say that in my 12-1/2 years withthe Foundation, 2006 has definitely been thebusiest for all of us. When I started recognizingspecific faces at Hartsfield Jackson Airport, Irealized I had truly spent a lot of time traveling. Wefinally came up for a breather during Thanksgivingweekend.As many of you know, we have held meetings allaround the country, including three via WebEx anda few focus groups. Topics have been varied andwe continue to reach out to the Gaucher communityfor suggestions on new topics. Please email uswith any ideas you may have. Dr. Pram Mistry isbecoming an expert at WebEx meetingpresentations and I would like to thank him for hiswillingness to be part of this technology which isnew for the Foundation. We have receivedexcellent feedback which helps us continue to learnand make these better. Thank you to everyonearound the country who helps with these meetings.Linda Rubenstein's “Duck Tour” is truly a favorite,and the Georgia Aquarium received rave reviews!Please check our web site for upcoming meetingsthroughout the year.I have had the privilege of being interviewed on tworadio shows this year, one with Jay Bernstein andone with Zev Brenner, which were dedicated toeducating the public about Gaucher disease.These two shows combined have a listeningaudience of about 60,000.September Awareness Month is growing, asevidenced by some of the meetings and events thatwere held this year: art exhibit, on-line auction, delithonand summit. We hope to continue to expandthis each year and are already in the planningstages for September 2007. If there is somethingyou would like to try in your area to promoteawareness of Gaucher disease, please let us know.We realize, through calls and inquiries, that ourawareness programs are working, but we alsorealize that we must continue our efforts.Page 4Rhonda P. BuyersExecutive DirectorNational Gaucher FoundationOnce again, successful clinics were held inTucson, AZ and Phoenix, AZ and manypatients were able to take advantage of Dr.Mistry's expertise.During 2006, the NGF has attendedconferences and been involved with meetingsof the American Jewish Press Association,the American Society of Human Genetics, theGlobal Organization for Lysosomal Diseases,the General Assembly and the JewishGenetic Disease Consortium. These allrepresent collaborative work we are doing inorder to further research, education andawareness, testing and up-to-date informationregarding Gaucher disease, as well as, alllysosomal diseases. We will continue to beinvolved, alone and collaboratively, to furtherthese goals.I would also like to take this opportunity tothank all of the individuals around the countrywho planned and worked on fund raisingevents. It was exciting to see how creativepeople were, as evidenced by the CarnivalDay event held in New York by the Tracy andTed Birkhahn family. The event raised about$2,000. Delaware Valley has held 11 verysuccessful golf tournaments with the sameindividuals planning and working each year.Without volunteers, events would not bepossible.May 2007 be filled with love happinessand good health!

By Michael G. MargolisReport From The World’s First Deli-thonJADAC, Jewish Associated Diseases Action Committeeheld its first annual Deli-Thon on September 14 in LosAngeles-area delis and featuring the world's largestcorned beef sandwich. The goal was to raise awarenessof Gaucher Disease, not money, in a setting that reachedsecular Jews and even non-Jews. More than 80 peopleattended the Deli-thon and all those attending emailed atleast five of their friends to inform them about GaucherDisease. Many greatly exceeded the five contacts andemailed most people in their computer address book. Insome cases, this exceeded over a hundred people. Allthose contacted were also asked to pass-on theinformation.The best news from the Deli-thon was the enthusiasticsupport from the delis. Owners and managers wereasked to allow us to place show cards on all their tablesfor the day of the event. Virtually all delis approached,easily accepted the offer to participate, many with muchenthusiasm. Jerry's Famous Deli committed their entirechain. In one case, at Factor's Deli, the manager thankedme for doing this work. I encountered two managersthat told me that Gaucher runs in their families.The support allowed the Deli-thon to take placethroughout Los Angeles. These Delis serve an averageof approximately 2000 people per day. With the 14delis who participated, approximately 28,000 peopleviewed the GD information show cards. In several casesthe delis left the show cards up for more than a day.They are still up in a few of the restaurants. It wouldhave been possible to include about another dozen delis,but we simply ran out of time.L.A. Television stations KTlA (channel 5), KNBC(channel 4 ), Fox News and KCAL (Channel 9) allwere very interested in covering the Deli-thon, but hadto put their stories on hold due to fires raging out ofcontrol on the outskirts of L.A, however, on November20th, Jewishjournal.com published a very nice storyfeaturing Gaucher disease and the Deli-thon.The Deli-Thon succeeded in several ways. The eventspread awareness outside religious organizations, (themajority of Jewish people are not affiliated with asynagogue), as well as the general public. The goodwillof the event also encouraged further participationfrom those religious and other organizations. Theevent served as a focal point to make initial contactwith the press. It is expected that future broadcastreports on Gaucher disease will go forward. Theenthusiastic participation suggests that a NationalDeli-thon is possible. I am confident that with theexperience we gained we could have a very effectivecampaign in all major cities with a high Jewishpopulation.Special thanks to the Delis who participated (below)and to Jewish Associated Diseases Action Committee(JADAC) , Genzyme, Ted Meyer and HeatherAvakemian for helping to make this wonderful eventpossible.Art's Deli, Studio CityJerry’s Famous Deli, Studio CitySolly's Deli & Bakery, Sherman OaksJerry’s Famous Deli, EncinoJerry’s Famous Deli, Woodland HillsBrent's Deli & Restaurant, NorthridgeWeiler's Deli, West HillsAbe's Deli & Family Restaurant, NorthridgeCanter's Delicatessen, Los AngelesGreenblatt’s, West HollywoodIzzy's Deli, Santa MonicaJunior's Delicatessen, Los AngelesJerry’s Famous Deli, WestwoodJerry’s Famous Deli, West HollywoodFactor's Famous Deli, Los AngelesHere’s the over 8-foot long Corned Beef on RyeAnd yes, we ate the whole thing!!Page 5

Page 6Suzanne’s Physical FitnessQ & A Corner1) Q: Although I receive enzyme replacement therapyevery 2 weeks for the past 4 years, 3 weeks ago Isuffered a bone crisis in my right ankle. The pain was sodebilitating. Now that the crisis has past, I feel so weak inmy thigh and ankle. Please advise me on someexercises I can do.A: Before beginning any exercise program I wouldsuggest you talking to your doctor first.The following are various exercises I would recommend.a) Knee Extension: sit on a firm chair, straighten yourright knee so that the leg becomes parallel to the floor,hold that position for 10 seconds then lower, thenstraighten your left knee and hold the position for 10seconds...repeat alternating legs for a total of 10repetitions for each leg.** to challenge your muscles use ankle weights (2-3lbs.)**b) Ankle Pumps: sitting on a firm chair, feet flat on thefloor, legs are hip width apart, rock on your heels as thesoles of your feet come up and hold the position for 5seconds then rock on the balls of your feet as your heelscome off the floor and hold for 5 seconds...repeat for 10repetitions...then perform 20 without the 5 second hold.c) Pillow Squeeze: remain sitting on the chair, feet flat onthe floor, place a pillow between your thighs, squeezeyour thighs into the pillow and hold the position for 5seconds ...repeat for 20 repetitions.d) Standing Calf Raises With Knees Straight: stand withboth feet straight and legs are hip width apart, knees arestraight, place hands on your hips (if balance is weak,then hold onto the wall or sturdy object), raise up on theballs of your feet as your heels come off the floor...repeatfor 20 repetitions or 2 sets of 10.e) Standing Calf Raises With Knees Slightly Bent: sameposition as above, this time your knees will be slightlybent, again raise up on the balls of your feet lifting yourheels off the floor...20 repetitions or 2 sets of 10.f) Calf Stretch With Knee Straight: stand and face a wall,place both hands on the wall, one leg is back with theknee straight and the foot flat and pointed straight ahead,the other leg is forward more towards the wall with theknee bent and foot flat on the floor, lean into the wall asyou feel the stretch in the back leg - make certain youkeep the heel down...hold the stretch for 20 seconds,then reverse your leg position and repeat the stretch...perform 2 sets.Suzanne Krupskas is a registeredphysical therapist since 1978. Shehas experienced multiple symptomsfrom Gaucher disease since beingdiagnosed in 1981. Suzanne is anavid speaker on Gaucher disease,and has written several articles on theimportance of exercise and physicaltherapy for those whom are living withthe disease. She can be reached bycallingThe NGF or by email:suzkrup@comcast.netg) Calf Stretch With Knee Bent: same position asabove, this time the back knee is bent, keep the footflat on the floor, lean into the wall and feel the stretchin the calf and the achilles tendon...hold the stretchfor 20 seconds, then reverse your leg position andrepeat the stretch...perform 2 sets.2) Q: My son is 12 years old. He was diagnosed withGaucher Disease at the age of 3. For the past 7years he's been receiving Cerezyme weekly.Preceeding treatments he suffered 2 bone crises inthe left hip. The crises have caused slight avascularnecrosis in the ball of the joint; and as a result helimps sporadically. With your expertise, would yourecommend my son to get involve in soccer? His gymteacher is encouraging him.A: No, I would not recommend soccer nor any otherimpact-type of sports. Your son already has anabnormality in one of the most important weightbearingjoints. So he's more vulnerable for furtherinjury. The two most important reasons your sonshould not get involved in soccer are:a) there's a greater chance for a stress fracture todevelop.b) the avascular necrosis may worsen.3) Q: Since your Q and A on osteoporosis (Spring2006 issue), I've been increasing my weight-bearingand weight-resistance exercises. I'm still involved in apool exercise program, but only 1 time weekly. Sixmonths ago I was going 3 times weekly. Amazingly Ifeel so much stronger now. I would like to diversifymy program by adding a Pilate class. Would that begood for the osteoporosis?A: Pilates is a great way to enhance bone density.There is a certain amount of resistance offered duringeach movement. Make certain you check out abeginners class and make the instructor aware ofyour osteoporosis. Remember, it is a fallacy whenpeople say '"no pain - no gain". Even the slightestresistance will assure an increase in bone density. Sonever push through the resistance.

Introducing National Gaucher Foundation CarePagesAs a service to the Gaucher Community, we are pleased to make CarePages availableto our members and visitors to our National Gaucher Foundation website.Connect, Share and Care!Now, you can make your own private, personalized web page for free. Learn more bygoing to our website at www.gaucherdisease.org.CarePages are private, personalized Webpages that allow individuals and their families to communicate and connect in a secureenvironment. This gives individuals the ability to create virtual communities of support.CarePages are free to individuals with Gaucher disease and their families and comply withall HIPPA privacy regulations. CarePages allow patient families greater control over communicationand provide an easy way for friends and loved ones to respond with messages of support.CarePages provides family and friends the opportunity to post updates, pictures, contactinformation and share stories with people in like circumstances. To access CarePages,individuals, friends and families visit www.gaucherdisease.org and click on the CarePageslogo. Help is available for the CarePages by contacting support@carepages.com or by calling 1-888-852-5521, Monday through Friday from 8 a.m. to 6 p.m., Central time.We encourage you to create your own website as part of your overall care and well-being.Have fun with it, have serious discussions, send good wishes, invite friends, family, co-workers,team members or religious leaders. Since you control content and access, you are the boss ofyour own virtual world. The support you receive from the community might just surprise you.Page 7

Dr. Robin A. Ely, PresidentMedical Director61 General Early DriveHarpers Ferry, WV 25425Ph: 800-925-8885 . Fax: 304-725-6429Email: ngf@gaucherdisease.orgOfficers:Robin A. ElyPresidentMedical DirectorMichael David EpsteinChairman of the BoardSecretaryNational Gaucher FoundationRhonda P. Buyers, CEOExecutive Director4106 Idlewood Parc CtTucker, GA 30084Ph: 800-504-3189 . Fax: 770-934-2911Email: rhonda@gaucherdisease.orgDennis BermanTreasurerBrian BermanBoard MemberFollowing is an overview of projects and activities in which theNational Gaucher Foundation has been involved during the year ended May 31, 2006.Research grants in excess of $180,000 have been funded through the NGF. This includes monies raised through the efforts ofour National and Delaware Valley Chapter Golf Tournaments. These funds helped to support research to find alternative treatments forGaucher disease.Educational meetings increased greatly and will be 32 in total by years end. These meetings provide much needed informationand give individuals with Gaucher disease, as well as their families, the opportunity to meet and interact with each other, often resultingin lasting relationships. These meetings and the relationships formed provide support systems not only to individuals who are already ontreatment, but to those who recently discovered that they, or their family member has Gaucher disease.Our English and Russian awareness programs have been successful in educating the lay and medical community and haveallowed the NGF to identify carriers and those who have Gaucher disease. The resultant effect has been our ability to help many moreindividuals be tested, diagnosed and treated. Clearly, without the benefit of these programs, many at-risk individuals would never bediagnosed.National Gaucher Awareness Month, held yearly in September, has allowed the NGF great flexibility in seeking additionalvenues for outreach, education and awareness. In addition to marketing opportunities through radio, magazines, newspapers andwebsites, an online auction is being held. A Summit Meeting in NY will discuss the need for more awareness of Jewish genetic diseasesand encourage input from key opinion leaders within the Jewish community. An art exhibit by Ted Meyer at the National Museum ofHealth and Medicine will become an event for outreach to a very diversified group and set the tone for future events such as HealthFairs.We would like to thank the many individuals who have raised monies for research education and awareness in such variedways, such as golf tournaments, 5K runs, carnivals and family events, and the sale of wristbands, CDs and pins.The NGF is happy to report that our CARE and CARE+PLUS programs have assisted more than 250 families and individualswith insurance premiums and ancillary expenses related to Gaucher disease. Barbara Lichtenstein has been dedicated to theseprograms since their inception and has brought them to their current levels of assistance.The continued growth of NGF's membership makes possible the production and printing of our quarterly newsletter and helps toprovide for various educational and collateral materials.GOLD (Global Organization for Lysosomal Diseases) has been very active this past year, and now has 136 memberorganizations, in 31 countries, representing all LSDs. The NGF has been involved since its inception and Rhonda Buyers has beenserving on the Management Council since 2004. If you would like to learn more about GOLD and its work, please visit the website atwww.goldinfo.org.The LSDN (Lysosomal Disease Network) was organized for the purpose of promoting and facilitating collaborative research,early diagnosis, and treatment of lysosomal diseases. It is comprised of researchers, clinicians and patient organizations. It will hold itsannual conference in Orlando, FL from December 7-9, 2006.The NGF has also been part of the (JGDC) Jewish Genetic disease Consortium since its inception in January, 2005. We arevery proud of the work that has been done to promote education and awareness of Jewish genetic diseases. We have beenrepresented at two major conferences, developed a Grand Rounds program, sponsored breakfast or lunch meetings at the AJP(American Jewish Press) conferences and worked to have specific testing included in various testing panels. Stay tuned for anotheryear full of activity.Sharon AdamsDirector, Meetings and Support Services61 General Early DriveHarpers Ferry WV 25425Ph: 800-925-8885 . Fax: 304-725-6429Email: sharon@gaucherdisease.orgRosina M. CristalliDirector, Community Relations4796 Kitty Hawk CircleGulf Breeze, FL 32563Ph: 800-541-7549 . Fax: 850-934-6129Email: rosina@gaucherdisease.orgCynthia J. FrankDirector of Development403 Main Street, #509San Francisco, CA 94105Ph: 877-649-2742 . Fax:415-839-5189Email: cyndi@gaucherdisease.org

National Gaucher FoundationA world of hope for families and individuals with Gaucher DiseaseSTATEMENT OF PUBLIC SUPPORT,REVENUES AND EXPENSESFOR THE YEAR ENDED MAY 31, 2006SUPPORT AND REVENUE:Support $2,298,930Revenue105,458TOTAL SUPPORT AND REVENUE $2,404,388EXPENSES:Program Services $2,383,005General and Administrative 110,671Fund-raising62,974TOTAL EXPENSES $2,556,650EXCESS/DEFICIT OF SUPPORTAND REVENUE OVER EXPENSES $ - 152,262FUND BALANCESBEGINNING OF YEAR $ 335,013FUND BALANCESEND OF YEAR $ 182,751Page 9

Special ContributionsJune 9 through November 9, 2006In Honor ofPage 10Benjamin Birkhahn by Wendy HartRob Biron's engagement by Mr. & Mrs. Arnold BerkowitzMakenzie Burkhart by Mindy BahlthJerry Byer's 50 Birthday by Nori KlarAlvin Canter's Birthday by Mr. & Mrs. Dick RadfordSol Catain's Birthday by Mr. & Mrs. Dick RadfordSol & Flory Catain's Anniversary by Mr. & Mrs. Dick RadfordJudy Dragin's Birthday by Mr. & Mrs. Mike WeissmanJanice & Jerry Dunn's new grandchild by Mr. & Mrs. Arnold BerkowitzMr. & Mrs. Gerald Farbman's Anniversary by Mr. & Mrs. Jay RogalCarol Fink's Birthday by Gerald ForquellMarianne Finke's Birthday by Joan FeldmanMichael Goldman by Jack Satter HouseRome Jaeger Goodman by Mr. & Mrs. Donald Bernstein and Sylvia CohenTillie Gordon's Birthday by Mr. & Mrs. Dick RadfordthHank Greenberg’s 75 Birthday by Harriet GreenbergCarole Helfand's Birthday by Mr. & Mrs. Dick RadfordJordon Hytken by Shayna SlebiodaAndrea Israel by Karen Rafsky and Karen IsraelBrady Jackson by Donna Clark, Ellen Corrigan, Tracey DuBois, Stephanie Jackson, SusanSirois,Judith Smart, Elizabeth StevensMike Joseph's Birthday by My. & Mrs. Jim KleinMara Kaliner's Birthday by Jessica ResnickCharles Kibler's Birthday by Mr. & Mrs. Jay RogalMichael Klar by Shirley KlarJudy & Jim Klein's Anniversary by Mr. & Mrs. David Kadish, Mr. & Mrs. Maynard Louis,Mr. & Mrs. Phillip ColemanShel & Jackie Kolner's Birthdays by Mr. & Mrs. Dick RadfordCarole Kushnir by Mr. & Mrs. William SavinDr. & Mrs. Alan Kushnir by Mr. & Mrs. Morton RothbergKen Lipkowitz by Bat Shalom HadassahAshlee Margolis' Birthday by Marcia WolfRobert Margolis by Emily WolfArthur Meisel's Birthday by Mr. & Mrs. Dick RadfordBernice Meisel's Birthday by Mr. & Mrs. Dick RadfordMandi & Wayne Meyerowitz's Birthdays by Mr. & Mrs. Stephen Cohen, Hilary Isakow, NancyPomish, Denise Sneag, Mary Schechter, Mr. & Mrs. Brian First, Mr. & Mrs. Arthur Klitofsky,Mr. & Mrs. Aubrey Meyerowtiz,Mr. & Mrs. Ian Smulowitz

Mr. & Mrs. Edward McGann's Wedding by Mr. & Mrs. Norman ZuckermanTed & Zelda Miller's Anniversary by Mr. & Mrs. Dick RadfordthMr. & Mrs. Gil Mintz's 50 Anniversary by Addison Delray Travel, Mr. & Mrs. GeraldCantor, Joan Eben, Mr. & Mrs. Lenard Glauber, Mr. & Mrs. Milton Katzman, Mr. & Mrs.Charles Krasny,Dr. & Mrs. Arnold Leibowitz, Mr. & Mrs. H. & G. Mendelsohn, Sylvia Pine, SusanShapiro,Seymour Spira, Mr. & Mrs. Norbert StraussIlene Oberstein's Birthday by Mr. & Mrs. Dick RadfordDanielle Patota by Pauline DolanMr. & Mrs. Dick Radford's Anniversary by Mr. & Mrs. Alvin CanterBeth Redpath by Sheree MoyerMuriel Rosenfield's Birthday by Dr. & Mrs. Wayne RosenfieldDr. Stein's Birthday by Mr. & Mrs. Dick RadfordRita Stein's Birthday by Mr. & Mrs. Dick RadfordRita & Hank Stein's children by Mr. & Mrs. Dick RadfordPeggy Sterling by Mrs. Lynne FisherLarry & Bea Tretter's Anniversary by Mr. & Mrs. Dick RadfordDominick & Julia Trombino by Fine Feathered Friends and Mr. & Mrs. JosephSalamoneNori Klar by Arlene SalterWilliam Weinlood's Birthday by Mr. & Mrs. Dick RadfordMike & Fran Weissman's Anniversary by Mr. & Mrs. David WeissmanLois Winograd's Birthday by Mr. & Mrs. Dick RadfordLois & Mel Winograd's Anniversary by Mr. & Mrs. Dick RadfordMelvin Winograd's Birthday by Mr. & Mrs. Dick RadfordSybil & Shelby Yastrow's Anniversary by Mr. & Mrs. Mike WeissmanSamuel & Ruth Young's Anniversary by Mr. & Mrs. Dick RadfordGabriel Zallik's Bar Mitzvah by Julie RachmanJoyce Zuckerman's Birthday by Barbara WeinbergLenny by Mr. & Mrs. Oliver ObstIn Memory ofJean Barr by Mr. & Mrs. Arnold BerkowitzJanet Bender by Eloise Epstein and Exxon Mobil ChemicalHarvey Berman by Mr. & Mrs. Lester Salisbury & FamilySanford Berman by Karen Bundy, Steve Blechman, Sally Woolsey, Liam Barker, Mr. &Mrs. David Hobbs, Janet TognoliniHailey Rose Diamond by Chris DiamondWalter Dunn by William Dickett, Mr. & Mrs. Joachim Grigull, Mr. & Mrs. Charles Palen,Mr. & Mrs. James Rosell, Lawrence Wallace, Edson Elementary School, UnitedAutomationJerry Fishman by Mr. & Mrs. Jim KleinDoris Flam by Mr. & Mrs. Morris Tulchinsky Page 11

In Memory ofJudy Hazan by Mr. & Mrs. David GervichHerbie by Dorothy WisotskyAnnette Karp by Mrs. Sharon LernerTerry Karp by Melissa KarpBarbara Klausner by Mr. & Mrs. Jim FerrandJean Klein by Mr. & Mrs. Norman FisherMartin Klein by Mr. & Mrs. Jeffrey Wechsler & FamilyIdele Kosofsky by Mr. & Mrs. Mark Blass, Mr. & Mrs. Manny Broady, Mr. & Mrs. Arthur Cohan,Gloria Davis, Mr. & Mrs. David Edovitz, Mr. & Mrs. Lenny Gross, Tammy Harris, Mr. & Mrs.William Jacobson, Mr. & Mrs. Michael Jusell, Mr. & Mrs. Steve Keenholtz, Kathleen Morreall,Mr. & Mrs. Melvin Pearlson, Mr. & Mrs. Jim Shuman, Samuel Silverman, Mr. & Mrs. MartyWolfGordon Levinson by Lynne FisherMartin Levinson by Ann LevinsonRabbi Marvin Needle by Mr. & Mrs. Morris TulchinskyHenry Newhouse by Mrs. Ruth NewhouseHarry Pollock by Mr. & Mrs. Robert Adler, Karen Amrein and Patrick Ratchford, TonyBobulinski,Leslie Goldfine, Andrew Haas, and Lori PerlowThelma Reisman by Barbara WeinbergAnthony Rossi by Mr. & Mrs. James CochranShirley Rudin by Mr. & Mrs. Harold GoldbergDeborah Saltz by Gail WallachRonald Schmitz by Mr. & Mrs. Arnold BerkowitzDorothy Siegel by Mr. & Mrs. Morris TulchinskyEsther Stavis by Mr. & Mrs. Stan GlanzbergPearl Sussal byMr. & Mrs. Jeffrey WolfSylvia Taffet by Mr. & Mrs. Morris TulchinskyHarold Unger by Mr. & Mrs. Marty Engel, Andrea Kolton, Mr. & Mrs. Raymond Padilla,Elaine Weaver by Mr. & Mrs. David GervichSam White by Mr. & Mrs. Ardeshir Bayat, Sharon Brender, Mr. & Mrs. Scott Denison, Mrs.Rickey Effron and Family, Mr. & Mrs. Neal Federman, Lareen Fender, Mrs. Jami FenderCronin, Mr. & Mrs. Steve Goldberg, Lynne Goldman Silbert, Trudie Goldstein, Kathy Harvey,Doreen Kates, Sarita May, Mr. & Mrs. Jerry Monkarsh, Lora Ricca, Shirley Rose, ArleneSarner, Mr. & Mrs. Dick Schulman, Mr. & Mrs. Karl Sussman, Mr. & Mrs. Saul Turtelbaum,Shammi Whitaker, Lisa Wicklman, Mr. & Mrs. Lee Willis,Mr. & Mrs. Ed Zalis, Mr. & Mrs. Robert MarxWe appreciate the Special Contributions to the National Gaucher Foundation on behalfof those who have donated in Honor of, or In Memory of a cherished loved one and paytribute to their honor and memory through publication of their names.Page 12

Join In on A Live Meeting Through TheLatest In Technology.....WebEx!The National Gaucher Foundation has made itpossible for anyone with a computer to be able toattend WebEx meetings.Through the many miracles of technology, WebExallows anyone to view, listen and participate inmeetings, conferences and seminars, without leavingthe comfort of their home or office.Whether a meeting is held in Los Angeles, or NewYork, you can participate in the meeting fromwherever you are. Go to our website atwww.gaucherdisease.org or check the list serve tosee what meetings will be held as WebEx meetings.This information will also be included in the meetinginvitations, which are sent out by the NationalGaucher Foundation.Prior to each WebEx meeting, the listserve, websiteand invitations will advise you of a phone numberand pass code which you will need to join themeeting. You will also be given instructions onwhat to do to log on, or any computer set-uprequired.We hope you will all enjoy the opportunity toparticipate in these meetings which would not, in thepast, have been possible for you to attend.$5.00 EachLittle People, Nurturing Women, Angels andLadies Red Hat Pins available.$14.00 eachIf you are interested in selling these pins to help the NGFraise funds for education and awareness of Gaucherdisease, please contact Sharon Adams at 800-925-8885or by email at ngf@gaucherdisease.org.New Audio and Video Postings on WebsiteNGF’s Fundraiser ItemsAvailable Throughout the YearNew Postings on NGF website:1) Ted Meyer art Exhibit “Scarred for Life” opening night of artExhibit, September 27, 2006 at the National Museum of Health andMedicine.2) Zev Brenner Radio Show: Interview with Rhonda Buyers,Executive Director of the National Gaucher Foundation.3) “ Know Your Heritage” - Provides various methods of makingnon-monetary pledges to help with education and awareness ofGaucher disease through program sign-up on our website. Allorganizations and individuals who pledge will be listed in ournewsletter and on our website.4) CarePages - a secure way of creating your own web page.Informative, fun and a great way to get your message out to familyand friends, post photos and give and receive messages of supportfrom around the world. Just click the CarePages logo to start.5) RSVP to Meetings or Register for an Event - an easy methodof communicating your desire to attend a patient meeting, aconference, a WebEx meeting, or any event in which you areinterested. Go to Homepage and click on Register.7) Language Translator - Yahoo Babel Fish will translate ourwebsite into most any language. Click on the icon at the top of thehomepage.8) Share Your Stories/Comments - Now you can read and shareyour stories or comments by clicking here, then submitting throughthe “click here” instruction at the top of the page.Coming soon: Interviews with Shalom USA Radio, AndreaTrombino and Rhonda Buyers and the NY Summit video highlights.NGF Fundraising Items: To purchase items in support ofthe National Gaucher Foundation and individuals withGaucher disease go to www.gaucherdisease.org and click onSupport the NGF.Wristbands: Engraved with, “Gaucher - Treat It & Beat It”.They are available for sale at our website atwww.gaucherdisease.org for $5.00 each. If you areinterested in selling wristbands in larger quantities, for afundraiser, please contact Cyndi Frank, NGF’s Director ofDevelopment at cyndi@gaucherdisease.org or call her Tollfree at 877-649-2742.Funds raised will help individuals withGaucher disease, through research, education and awareness.Purchase a music CD “ Piano Reverie”, by Dr. KenLipkowitz. Contact Sharon Adams directly at 800-925-8885or email her at NGF@gaucherdisease.org. $20 per CD. Todate, hundreds have been sold. You can hear a sample of Dr.Lipkowitz’s musical abilities by watching the Ted MeyerExhibit opening night video.And don’t forget our “ Little People” pins shown here.Page 13

Duck Tour for Gaucher PatientsBy Linda RubensteinLinda Rubenstein andPhillip the catererWelcome to Boston Duck Tours!You've never toured Boston in anything that comes close to Boston Duck Tours.The fun begins as soon as you board your "DUCK", an authentic, renovated WorldWar II amphibious landing vehicle. First, you’re greeted by one of our legendaryConDUCKtors, who narrates your tour. Then you're off on a journey like you've never had before. You cruise byall the places that make Boston the birthplace of freedom and a city of firsts, from the golden-domed StateHouse to Bunker Hill and beyond.On Sunday, September 17th, that is just what everyone was treated to after their Boston GaucherMeeting. Everyone with children was encouraged to come and bring the kids. They love the DuckTours!!The day started at 10:30 AM, then Dr. Grabowski who is a pediatrician and Geneticist spoke to theaudience, followed by Shelley Holt who is a Home Care Infusion Nurse. She was great to speak andwas available to answer any questions. The meeting was held at the new Genzyme Center and afterthe speakers, there was lunch and tours for all participants.After lunch the Duck Tours came to pick us up at the Genzyme Center. This was a great opportunityfor all the parents with kids to meet each other, get questions answered and have lots of fun. My sonwas there...he still talks about the last Duck Tour (about 4 or 5 years ago) and the girl with Gaucherdisease, he met there. He was hoping to see her again!We appreciate the fine speakers, the opportunity to ask questions of the speakers, the chance to seeone another and see the Genzyme Center and we had, not only a very educational and worthwhileexperience, we had a wonderful time! Check out Duck Tours in your area. They are great for just afew people or a crowd of people and really give individuals something to look forward to aftermeetings.Golfing Fore a Great Cause for 13 YearsWachovia Golf ClassicThe Wachovia Golf Classic was held at the TournamentPlayers Club at Avenel in Maryland on August 14, 2006.Through sponsorships from Wachovia, Capital One, AmicusTherapeutics, Shire Human Genetic Therapies, Nova Factor,Genzyme and Caremark, the Tournament was a greatsuccess. Over a hundred golfers came out to enjoy thewonderful day of golf on one of the most exclusive coursesin the US. The day's events were capped off by a 19th HoleReception, where everyone gathered for great food andprizes. The Tournament raised over $75,000 for Gaucherresearch, education and awareness!Each year the NGF is fortunate to have the support of manyfriends who work tirelessly to acquire sponsors and golfers.To all the golfers and volunteers who lend their support eachyear, we say Thank You. The success of the event is a directresult of your efforts and we could not have done it withoutyou.Page 14Delaware Valley ChapterGolf Fore Gaucher Disease ClassicEach year, the Delaware Valley Chapter's GolfFore Gaucher Disease Classic gets bigger andbetter. This year, through the hard work of Larryand Lois Kaliner, Shirley and Harold Goldbergand Lynn and Gary Feuer, the Golf Tournamentwas one of the most successful in its 11 yearsrunning.On October 13, 2006, over 80 golfers enjoyed awonderful autumn day of golf, great prizes andcamaraderie. The tournament culminated withan evening dinner reception where golfers andfriends enjoyed a silent Auction, raffle drawingand awards presentations. The event raisedover $80,000 for Gaucher research andeducation! The NGF would like to extend aheartfelt Thank You to the Kaliners, Goldbergsand Feuers and their committee for all their timeand effort to make the tournament successful.

Because We Care About You and Your Family....Financial Assistance Programs Are Available forIndividuals With Gaucher DiseaseCare and Care+Plus ProgramsThe National Gaucher Foundation, Inc. sponsors two financial grant programs to help families and individualswith Gaucher Disease to pay for certain Gaucher-related expenses. To be eligible for either program, an applicantmust: a) Have Gaucher Diseaseb) Demonstrate extraordinary financial hardship for eligible Gaucher related expensesCARE PROGRAMThe CARE Program grants funds to subsidize, or to purchase in full, a health insurance policy (primary, secondaryor both), to which the patient is entitled or eligible, but for which the patient does not have adequate personalresources to enroll.CARE+PLUS PROGRAMThe CARE+PLUS Program provides financial assistance to eligible Gaucher patients for a variety of Gaucherrelatedexpenses, such as diagnostic tests, infusion charges, travel expenses, and over-the-counter medications.The program excludes deductibles and co-pays.Both programs involve an application process and review by the Care Board for determination offinancial and medical necessity.The National Gaucher Foundation realizes that Gaucher patients have extraordinary needs that may cause afinancial burden on themselves and their families. These two programs have been developed to lighten this burden.For additional information contact Barbara Lichtenstein, Program Director of the CARE and CARE+PLUSOur Heritage and Our Health MeetingsTo be held in January, 2007.Health-Related Meetings Not Sponsored by the NGFMonday, January 8th , 2007 at 10:00 am at theAlpert Jewish Community Center. This meeting willpartner with an NGF patient advocate.Go to www.gaucherdisease.org to see other “OurHeritage and Our Health” meetings. Click onNews, Events and Meetings, then click “Non-NGFSponsored Health-Related Meetings” for moreinformation about these meetings.Thursday, January 11th, 2007 at 6:30 -8:30 atJewish Community Center of San Francisco. GaryS. Frohlich, MS, CGC and Cyndi Frank, Director ofDevelopment for the NGF, present “Our Heritageand Our Health” with a focus on Gaucher Disease.Saturday, January 27th, 2007 at 7:00pm- 9:30pmat Congregation Beth Am, Los Altos Hills, CASunday, January 28th, 2007 at 10:00-11:00am atCongregation Etz Chayim in the Beit Knesset andTuesday, January 30th, 2007 at 7:30-9:00pm atTemple Beth Sholom. Santa Ana, CA.Online Discussion Group - Join Today!We’d like to invite you to join the list serve if you, your familyor friends has any rare metabolic diseaseIn 1994, Wayne Rosenfield started an online discussion group forpeople who have Gaucher Disease or other rare inheritedmetabolic diseases. The discussion group has well over twohundred subscribers throughout the world. It includes people withGaucher Disease, family members, doctors, nurses, researchersand others interested in Gaucher Disease or other inheritedmetabolic disorders. It's accessible twenty-four hours a day and itis independent of any entity’s control.This discussion group is a means of networking andcommunication for families and individuals with Gaucher disease.They are able to share experiences and opinions, and providesupport to each other. The NGF encourages everyone who hasaccess to the Internet to join the online discussion group. TheNGF has instructions on its website at:www.gaucherdisease.org/list.htm on how to join the list.Remember, this discussion group is not just for those individualsconnected with Gaucher disease and we hope that you willbecome a part of it and share information with others and join indiscussions. Page 15

National Gaucher Foundation Web HitsAs of November 22, 2006Page 16Research:New York University School of MedicineNew York, NYOral Therapy Being Developed for theTreatment of Gaucher DiseaseThe New York University School of Medicine,Division of Neurogenetics, has been selected toparticipate in a worldwide clinical study to test a neworal medication for the treatment of patients withtype 1 Gaucher disease. The investigational drug iscalled Genz-112638 and it has been previouslytested in three Phase 1 studies in normal volunteers.This Phase 2 study is designed to assess the serumconcentrations of Genz-112638 in patients with type1 Gaucher disease and to determine whether thedrug shows to be safe and effective treatment forpatients with the disease.We would like to inform you that NY University iscurrently enrolling patients in this study, beingconducted by physicians with expertise in treatingpatients with Gaucher disease.As you know, the current standard of care forpatients with type 1 Gaucher disease is enzymereplacement therapy (ERT) with recombinant humanglucocerebrosidase (CerezymeÒ [imiglucerase forinjection)). Cerezyme is usually administeredintravenously, every two weeks.Genz-112638 is a new oral medication that functionsby decreasing the synthesis of glucosylceramide,the substrate that accumulates in the lysosomes ofpatients with Gaucher disease. Go towww.gaucherdisease.org and click on Research toread the whole article on this important research andtrials.For more information please contact the primaryinvestigator at NYU, Dr. Maryam Banikazemi at212 263 8344 or maryam.banikazemi@med.nyu.eduUnique Visitors = 36,094Hits to the site = 610, 733We wish to thank all of our visitors andappreciate your interest in visiting ourwebsite at www.gaucherdisease.orgVisit the National GaucherFoundation web site oftenKeep up with the latest news and events, research,meetings, and all of the interactive things to do and see,such as creating your own webpage, making a pledge tohelp with the “Know Your Heritage” program, watchingvideos and hearing audios, sharing and reading storiesand comments from other individuals who share yourinterests, RSVP to pateint meetings and conferences, andmuch more!Coming to a City near you…By Sharon Adams, Director of Membership and Services,National Gaucher FoundationFrom Los Angeles and San Francisco, CA toDenver, CO and Chicago, IL to Atlanta, GA andBoston, MA, the NGF held over 30 Gaucher advocacymeetings in 2006. We are thankful to the guestspeakers who took time out of their busy schedules toparticipate in these regional meetings. Topics ofdiscussion included fatigue, bone issues, diseasemanagement and new research developments. If youare interested in planning a meeting in your area in2007, please contact the NGF at (800) 925-8885.Meeting information is always available on theNGF's website at www.gaucherdisease.org, underNews & Events. The Events Calendar is regularlyupdated so we encourage everyone to visit the siteperiodically to keep abreast of meetings and eventsaround the country.National Gaucher Foundation DirectoryPh: 800-GAUCHER . Fax: 304-725-6429Robin A. Ely, PresidentMedical DirectorPh: 800-925-8885email: robinbmd@aol.comRhonda P. Buyers,CEOExecutive DirectorPh: 800-504-3189email: rhonda@gaucherdisease.orgSharon Adams, DirectorMeeting and Support ServicesPh: 800-925-8885email: sharon@gaucherdisease.orgRosina Cristalli, Marketing DirectorPh: 800-541-7549email: rosina@gaucherdisease.orgBarbara Lichtenstein, Director Care and Care+PlusPh: 866-346-8176email: blichtenstein@comcast.netCynthia J. Frank, Director of DevelopmentPh: 877-649-2742

What if the National Gaucher Foundation earned a penny everytime you searched the Internet? Well, now we can!GoodSearch.com is a new search engine that donates half its revenue, about a penny per search, to the charities itsusers designate. You use it just as you would any search engine, and it's powered by Yahoo!, so you get great results.We know a penny doesn't sound like much, but if we had 100 folks searching the internet just twice a day, it wouldtotal $730 a year, or 500 of us searching four times a day, it would raise about $7,300 a year without anyonespending a dime!Just go to www.goodsearch.com and be sure to enter the National Gaucher Foundation as the charity you want tosupport. You can make GoodSearch your homepage and you can download the GoodSearch toolbar. Whatever youneed to remind yourself to use GoodSearch each time you search for something on the Internet.GoodSearch will feature the NGF as the Charity of the Day on January 2, 2007As you know, the National Gaucher Foundation has always encouraged sharing their stories on our website orin our newsletter, as we never know whose life will be touched and who we help through sharing. The NGFgets hundreds of phone calls in a month, or in a quarter, or in a year. Below are exampIes of stories that arenot only uplifiting for the NGF, but might be uplifting to others, as well. Go to www.gaucherdisease.org andclick on “Stories/Comments” to share a story with others.#################I wanted to tell you about a phone call I had today with a man in Las Vegas who is 91 years old, has Gaucherdisease and not on ERT. We had such a great conversation. He is mildly affected; gets his blood countschecked regularly. I asked him to write an article for our newsletter about his personal story with Gaucher. Heis the oldest person that I know of, and have talked to, with Gaucher disease. He has been followed over theyears by Dr.Ernest Beutler and Ari Zimran.He asked me if I thought he has lived a long time, despite having Gaucher! I told him that he has lived a longtime for anyone! Most people don't live into their 90s. Anyway, I just wanted to share the call with you. It wasvery Uplifting!Sharon AdamsDirector of Membership and ServicesNational Gaucher Foundation#################I attended the genetics conference yesterday at the Jewish Community Center of Greater Boston. I wasthe Gaucher representative for a 5-person discussion panel at the end of the day. I spoke second, whichwas a good thing, considering what the other people had to say. My chronology of serious Gaucher-relatedproblems ends abruptly in 1993 when I began ERT. My story seems positively joyous compared to peoplewho have placed a child in a nursing home, are doing tube feedings for their children at home, or havewatched their babies deteriorate and die. Out of sight from the audience, the Mom representing Tay-Sachs had a photograph on the table in front of her of her beautiful baby girl. She said that, when herhusband returned from the bedside of their niece at the time of her death, he brought her the news thatthe death part of the disease is not so bad, and "It will be alright."I'm sharing this with you because I feel a little guilty that the Gaucher story I related was so upbeat. Myown Gaucher experience just gets worse and worse, until suddenly the bad things stop. There are so manyother disorders that are causing people to suffer right now.I think we can take some pride in pioneering the first of these disorders to have effective treatments.Wayne Rosenfield, Ph.DPage 17

From the Development Office…By Cyndi FrankGoodSearch.com is a new search engine that donates half its revenue, about apenny per search, to the charities its users designate. You use it just as youwould any search engine, and it's powered by Yahoo!, so you get great results.Please see our GoodSearch article on page 13.Be sure to spread the word! The more people who use GoodSearch, the moremoney that can be raised for Gaucher disease. The sky is the limit! Just go towww.goodsearch.com and be sure to enter the National Gaucher Foundationas the charity you want to support. You can make GoodSearch yourhomepage and you can download the GoodSearch toolbar. Whatever youneed to remind you to use GoodSearch each time you search for somethingon the Internet.Update 11/22/06: Note to Genetic Disease list serveHello all,I've been watching the numbers grow with usage on the GoodSearch site. Asyou've discovered it works the same as any other search engine, and itreally is a great way to raise money for Gaucher disease without spending adime! Once we reach $100 the NGF will get its first donation check. I'llkeep you posted and let you know when that is.Thanks so much for being so quick to respond to this and helping to raisemoney for Gaucher. I hope you keep using it! Cyndi FrankAs you know, we rely on donations from individuals like you to help us carry out ourmission of finding a cure for Gaucher disease through research, awareness andeducation. We greatly appreciate your generous donations, bidding on auction itemsin the online Gaucher Awareness Auction, and participating in local fundraisers in yourhome towns. This year our supporters have had great success with a variety offundraisers to support Gaucher, from holding carnivals, golf tournaments and 5KRuns/Walks to selling Gaucher wristbands and Little People pins at schools andcongregations, and even through something simple like a company matching donationat your workplace. Thank you for a successful year of fundraising! With continuedgrassroots efforts like this, we'll be able to raise money and awareness of Gaucherdisease and all those who suffer from Gaucher and their families will benefit.If you'd like to help by holding a fundraiser, please contact Cyndi Frank at 877-649-2742 or Cyndi@gaucherdisease.org to talk about ways you can help. Together wecan come up with a viable and creative way to benefit Gaucher disease.Thank you for your continued support. Wishing you a wonderful and healthy holidayseason!Please contact Cyndi Frank, Director of Development, at 877-649-2742 orCyndi@gaucherdisease.org if you would like to help the NGF with fundraising efforts.To make a gift or donation, please contact Sharon Adams, Director of Membershipand Services, at 800-925-8885 or ngf@gaucherdisease.org.Remember, all donations and out-of-pocket expenses used toward holding afundraising event are tax-deductible under section 501(c)(3) of the IRS Code.Page 18Cyndi FrankDirector of DevelopmentNationalGaucher FoundationThank you GaucherCommunity and Beyond!Our first ever online GaucherAwareness Auction was agreat success! The auctionraised over $17,000, andwe're thrilled with the results.We hope you had theopportunity to participate inthe fun of online bidding, andwe hope you found that itwas a meaningful way tosupport Gaucher disease.Many thanks to ActelionPharmaceuticals, our leadsponsor for the event, and allthose who participated in theauction, particularly ourdonors who offered thewonderful items that wereavailable in the auction.Please see auction donors onpage 15. Without them, thisauction would not have beenpossible.

Auction Donors:Actelion PharmaceuticalsAmicus TherapeuticsAnonymousStacy Blackman ConsultingSteven E. Brown, Ph.D.Cinta SalonCosentino Signature WineriesBeth Goozman ElkisRose-Lynn FisherLois Ellen FrankNeil & Maureen GoldGunlach Bundschu WineryKatherine HelmondJames Everett Johnson “JJ”King Features SyndicateFlorence LewisNan LowittMacy'sTed Meyer/Art Your WorldMumm Napa WineryPhotography by HyettLois RikerKaren Shaff/Beads of LightSonoma Valley Film SocietyShelley VivianiKen WolfYountville InnAcuity Vision BoutiqueAnonymousBell Wine CellarsThe Bowen FamilyCeja VineyardsMarcia CooperCupcake NationBridget EngelDana & Steve FlachGary S. FrohlicheGoode RiderLouise & Alan HallamInn of the AnasaziBruce Lloyd KatesCarole & Alan KushnirKen LipkowitzMostly BearsGigi McMillin, RNCMovado Group Inc.Pacific Blues CaféJonathan PollackWayne RosenfieldSix Flags Marine WorldToesies by CariDawnBarbara WiednerWoodhouse Family CellarsLois Zetter/The Zetter CollectionPlease join us again next year for the 2nd annual online Gaucher Awareness Auction duringthe month of September. If you'd like to serve on next year's auction committee to help raisemoney for Gaucher disease, please contact Cyndi Frank at 877-649-2742 orCyndi@gaucherdisease.org. Committee members will help to procure items for the auctioncatalog, make contacts for corporate sponsors and get the word out for the auction. We plan tohave the maximum amount of people online bidding so that we can raise even more money tofind a cure for Gaucher Disease by funding vital research programs, meet the ever-increasingneeds of patients and families and promote community/physician awareness and educational programs.September, 2007 is National Gaucher Awareness MonthSave the Date for A National “Know Your Heritage” Summit in September.June, 2007 “Know Your Heritage” WebEx Meeting for all Jewish Community leaders, Jewishorganizations, health providers, geneticists, individuals and families affected by these diseasesNovember, 2007 National Museum of Health and Medicine - Art Exhibits by individuals withrare Jewish genetic diseases.More News in our next issue.Page 19

What a grand opening night it was! Ted Meyer’s“Scarred for Life” exhibit hit a home run withthe audience, the media and art criticsthroughout the country.Ted’s exhibit and opening night were covered by theNew York Times, Richmond Times Dispatch,Washington City Paper, the Washington Post, toname just a few of the articles written.Held at the National Museum of Health andMedicine in Washington, DC, Ted’s art exhibit wasa perfect fit among several different venues,mediums and philosophys.The wonderful relationship that developed betweenthe artist, the Museum, the National GaucherFoundation, the NIH and everyone involved, couldnever have been planned, nor imagined.For more information about the “Scarredfor Life” artwork, you can contact Ted andask him for a catalog of his entire body ofwork which is now on display at theMuseum until March, 2007. You can emailhim at: ted@artyourworld.com.The NGF is looking forward to the possibility ofanother event at the Museum in 2007 and will keepyou up-to-date as we get through the planningstages. As a result of our relationship, the NationalMuseum of Health and Medicine has extended to usthe opportunity to participate in four Health Fairs.They hold one each month and we are thrilled to beable to participate.A lot of people were involved in the planning andexecution of the opening night event, as well as theexhibit. In a wonderful and moving speech, TedMeyer extended his thanks to JADAC, Genzyme,the National Museum of Health and Medicine andthe National Gaucher Foundation.Page 20Ted Meyer, Britt Oiulfstad, Paula GrahamA highlight of the evening was Dr. Roscoe Brady’sspeech about wiping out genetic diseases, perhapsin our lifetime.You can see the entire opening night event at ourwebsite at www.gaucherdisease.org. The video waswonderfully shot and edited by Jay Chapin ofDreamStar Productions. Oh, and we must notforget, the food and desserts were great. Dr. KenLipkowitz’s piano performance was wonderful andthe entire evening was a bit magical.Thank you, Ted for your art, your passion andfor helping to bring us all together.

Ted MeyerAnne Meyer, Britt Oiulfstad and Rhonda BuyersMary Nathan, Rosina Cristalli, Ted MeyerPage 21

More Photos from Opening Nightof the Ted Meyer,“Scarred for Life” ExhibitSome of the artwork displayed at the TedMeyer exhibit held at the National Museum ofHealth and Medicine. Runs until March, 2007Adrianne Noe,Ph.D, DirectorNational Museum of Health and MedicineDr. Ken Lipkowitz plays piano atopening night of the exhibit.Ken Wolf and Barbara Lichtensteinshare a photo opportunity.Page 22As one of the guest speakers, Dr.Roscoe Brady speaks about hishope for wiping out many geneticdiseases.Terri Meehan, Ken Wolf, A.J. Joshi, Chuck YasenkaTo see learn more about the “Scarred for Life”exhibit or the National Museum of Health andMedicine, go towww.nmhm.washingtondc.museum/ or call(202) 782-2200.To see more artwork by Ted Meyer, go to hiswebsite at:http://www.artyourworld.com/home.htm

Jewish Genetic Disease Consortium AttendsThe 75th UJC General Assembly in Los AngelesRecently published by Jewishweek.com, this article is a wonderful tribute to how far the JGDC has come in one year. Thearticle, entitled Genetic Package Deal was published on October 24th and written by the health writer, Francesca LunzerKritz - Health Writer. Francesca begins with the following: By joining forces, groups addressing Jewish hereditarydiseases are able to increase awareness and prevention. Go tohttp://www.thejewishweek.com/bottom/specialcontent.php3?artid=1291 to read the entire article. It is also posted at theJGDC website and the NGF website.The UJC event, which began on November 12th, included inspirational leaders from around the world to kick off thisunique GA. Israeli Foreign Minister Tzipi Livni, Los Angeles Mayor Antonio Villaraigosa, Zeev Bielski, Karnit Goldwasserand Fentahun Assefa-Dawit were joined by artists Debbie Friedman, Mare Winningham, Jon Voight, Jeff Goldblum, MikeBurstyn, Dave Koz, Dan Gordon and Julie Silver for an afternoon of music, hope and unity.Additionally, UJC's premiere event at the L.A. Convention Center brought more than 5000 Jewish leaders together underone roof and was a perfect venue for the JGDC to have an education booth, disseminate information on all Jewish geneticdiseases and discuss their successful pilot screening program.On Wednesday, November 14, participants attended a conference on “Challenges and Opportunities: Israel 2006”. Twoof the Jewish State’s most dynamic and innovative leaders discussed post-war Israel and how the Israeli people arelooking to the future. Speakers included: Isaac Herzog, Israel Minister of Tourism; Yuli Tamir, Israel Minister of Education;Rand Harris, President, Los Angeles Zimriyah Chorale, Israeli Prime Minister Netanyahu, and Prime Minister EhudOlmert.The JGDC had an opportunity to meet with various community leaders to discuss how to utilize the Jewish community tohelp educate their constituents, congregants, individuals, the medical community and organizations about these diseasesand how many lives are touched by their affects. It is estimated that 1 in 4 Jewish people of Ashkenazi ancestry inheritsthe carrier gene for one of the diseases listed below.The Jewish Genetic Disease Consortium has become the largest supporter of screening and testing for these diseasesand they have had great success already with their pilot programs in Pittsburgh, Philadelphia and Boston. Additional fundswill be needed to make this successful program available in all major cities populated by individuals of Jewish ancestry, aswell as continuing its campaign for education and awareness.The Screening Program is headed by Dr. Adele Schneider, Director, Victor Center for Jewish Genetic diseases at AlbertEinstein Medical Center, Philadelphia and Susan Gross, MD, FRCS(C), FACOG, FACMG, Associate Professor and codirector,Department of Obstetrics & Gynecology and Women's Health Montefiore Medical Center/Albert Einstein Collegeof Medicine, Philadelphia, PA.The JGDC represents a dynamic community based source that can help create a nationwide screening and testingprogram which allows for testing at an affordable rate.Visit www.jewishgeneticdiseases.org for a wide variety of informationabout the following Jewish genetic diseases and its 15-member alliance.Bloom’s Syndrome . Canavan Disease . Cystic Fibrosis (CF) . Familial Dysautonomia (FD)Fanconi Anemia Type C . Gaucher Disease Type 1 . Glycogen Storage Disorder Type 1a (GSD1a)Maple Syrup Urine Disease (MSUD) . Mucolipidosis IV (Ml4) .Niemann-Pick Disease Type A . Tay-Sachs DiseaseJewish Genetic Disease Consortium315 West 39th Street, Suite 701 . New York, NY 10018(866) 370-GENE (4363)Info@JewishGeneticDiseases.orgPage 23

National Gaucher FoundationMedia for 2007See our Gaucher ads in:English Media:Hadassah MagazineMoment MagazineJewish WomanNew VoicesHeebB’nai B’rith NationalNew York Jewish WeekPhiladelphia Jewish WeeklyUnited Synagogue ReviewAmerican Jewish SpiritWorld Jewish DigestFL Jewish DirectoryGuiide to Jewish PhilanthropyRadio: Shalom USA & Zev Brennerand Barry Reisman Show (Phila.)Russian Media:Russian TV guideHealth MagazineYevreiski MirNovom SveteReklama ChicagoKurier East CoastKurier Los AngelesPanoramaVoice of Israel(Some media is subject to change)The National Gaucher Foundation is anon-profit, tax exempt organizationfounded for the purpose of encouragingsupport and raising funds to promoteresearch into the causes and cure ofGaucher Disease. Funds will bespecifically directed to scientific,educational or literary purposes in keepingwith a charitable foundation.In This Issue2 & 3 Dr. Roscoe Brady Tribute4 End of Year Letter fromRhonda Buyers5 Gaucher disease Deli-Thon6 Suzanne’s Physical Fitness Q & A7 Make Your Own website withCarePages8 & 9 Overview of NGF Projects andFinancial Statement for 200610-12 In Honor of and In Memory Of13 NGF Fundraising Items andNew Postings on Website14 Duck Tour Meeting in Boston andWrap-Ups of Golf Tournaments for ‘0615 Information on CARE and CARE+Plusand Online Discussion Group16 Research on Genz-112638Oral Therapy Trials17-18 News From New DevelopmentOffice on Fundraising Programs19-22 Opening Night of “Scarred for Life”Ted Meyer Exhibit at National Museumof Health and Medicine23 Jewish Genetic Disease ConsortiumWrap-Up of UJC convention in L.A.The Newsletter attempts to report all items of interestrelating to Gaucher Disease. We will not willinglyreproduce inaccurate or libelous material. Anytreatment, therapy or institution that is recommended inthe newsletter should be discussed with an individual’slocal physicians. Neither the NGF nor contributingauthors take responsibility for such recommendations.The editor reserves the right to make corrections as areappropriate and in accordance with established editorialpractice in material submitted for publication.National Gaucher FoundationNational Gaucher Foundation . 61 General Early Drive . Harpers Ferry, WV 25425 . 800-GAUCHERFor more copies of this newsletter contact us at ngf@gaucherdisease.org, or go to our www.gaucherdisease.org,click on “News and Events”, then on NGF Newsletters for a PDF of this issue.