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Contents The Genetic Connection ......... 4 In Your Corner Patient Education Liaisons and Genetic Counselors ......... 7 Seven Tests to Determine the Best Treatment for <strong>Gaucher</strong> Disease ............... 13 Talking to Siblings of Children With <strong>Gaucher</strong> Disease Type 1 .... 15 Understanding the Progression of <strong>Gaucher</strong> Disease Type 1 ...... 18 Foreword When parents face the diagnosis of <strong>Gaucher</strong> disease type 1 in one of their children, naturally their concerns become focused on the newly diagnosed child. However, it’s just as important to discuss a child’s <strong>Gaucher</strong> disease type 1 with his or her siblings, whether the diagnosis comes as a surprise or whether it is already established by the time a sibling is born. Brothers and sisters play a key role in understanding what it means to live with <strong>Gaucher</strong> disease type 1 in the family. That’s why this issue contains an article devoted to Talking to Siblings of Children With <strong>Gaucher</strong> Disease Type 1. After all, <strong>Gaucher</strong> is a family disease, inherited from parents or grandparents. Learn more about the Genetic Connection of <strong>Gaucher</strong> disease type 1 on page 4. This article explains how the genetic mutation for <strong>Gaucher</strong> disease type 1 may be passed from generation to generation, and how a Genetic Counselor or a Patient Education Liaison may be able to help couples who are carriers or who have <strong>Gaucher</strong> disease type 1, to understand the reproductive risks. A Patient Education Liaison, not only helps educate couples and families so they know who is at risk and to understand who could benefit from testing, but he or she is committed to helping the entire family deal with <strong>Gaucher</strong> disease type 1. This issue features the article, In Your Corner: Patient Education Liaisons and Genetic Counselors. These Counselors are often the first people to explain what a <strong>Gaucher</strong> disease type 1 diagnosis can mean, in very real terms. They are the initial educators, helping patients with <strong>Gaucher</strong> disease type 1 to understand how the disease progresses and what tests may be involved. Articles in this issue’s <strong>Horizons</strong> will also help readers to understand the progression of <strong>Gaucher</strong> disease type 1; and to learn about the many tests that assess the status of the disease, evaluate the effectiveness of an existing treatment plan, or determine the important next steps in therapy. An educated patient helps to shape his or her own horizon. As with every issue of <strong>Horizons</strong>, we would love to hear your comments and feedback, so don’t hesitate to send us a note. We look forward to hearing from you. —Your team at Genzyme Cerezyme ® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of type 1 <strong>Gaucher</strong> disease that results in one or more of the following conditions: anemia (low red blood cell count), thrombocytopenia (low blood platelet count), bone disease, hepatomegaly or splenomegaly (enlarged liver or spleen). Important Safety Information Approximately 15% of patients have developed immune responses (antibodies). These patients have a higher risk of an allergic reaction (hypersensitivity). Use Cerezyme ® (imiglucerase for injection) carefully if you have had an allergic reaction to the product in the past. Symptoms suggestive of allergic reaction happen in 6.6% of patients, and include anaphylactoid reaction (a serious allergic reaction), itching, flushing, hives, an accumulation of fluid under the skin, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. Side effects related to Cerezyme administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported side effects include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Because Cerezyme therapy is administered by intravenous infusion, reactions at the site of injection may occur: discomfort, itching, burning, swelling or uninfected abscess. Cerezyme is available by prescription only. For more information, consult your physician. Please see accompanying full Prescribing Information on pages 11-12. Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit FDA.gov/medwatch, or call 1-800-FDA-1088. Please see accompanying full Prescribing Information. Winter <strong>2011</strong>/2012 / <strong>Horizons</strong> 3
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