Gaucher Community News - National Gaucher Foundation

WORLD Symposium 2011
By Rhonda P. Buyers
The WORLD Symposium has become the
annual meeting to attend for representatives of
the Lysosomal Storage Disorders (LSD) world.
Researchers, physicians, patient organizations,
parents, patients and pharmaceutical companies
were in attendance at this meeting during February
16th through 18th, in Las Vegas, NV.
Sessions ran from 8:00 a.m. to 5:00 p.m. each day,
with a total of 70 presentations given by many of
the most well-known experts in the LSD field. As
to the topic of Gaucher disease, some of the take
home messages for the Gaucher community were:
- More researchers are working on the
Gaucher/Parkinson connection;
- After two years, eliglustat tartrate (the
oral drug now in clinical trials by Genzyme)
has proven to be safe and efficacious and
radiologic monitoring demonstrated progressive
improvement of Gaucher disease Type 1 bony
manifestations;
During the meeting, an announcement was made
regarding Genzyme’s sale to Sanofi-Aventis SA.
Many Genzyme employees were in attendance,
so I had the opportunity to meet and discuss
with them the impact this would have on the
Gaucher community. I was reassured, at all
levels, including by John P. Butler, President,
Personalized Genetic Health, that the Gaucher
community would see no difference in the
excellent service it has experienced over the
years--patients will remain the priority.
Also, relationships will remain as they have been.
I attended a meeting with Genzyme in which we
were given an update on the new Framingham
plant and reassured that everything possible is
being done to insure a drug shortage such as the
one in 2009 will not reoccur.
Additionally, I had the opportunity to meet with
representatives from several other Lysosomal
Storage Disorder groups. Patient Organizations
were in attendance from as far away as the UK
and New Zealand.
One of our goals as a group was to determine
what role we play in the process of determining
research projects that are presented to the NIH
for funding, as the NIH mandated that the Patient
Organizations’ support be an integral part of this
process. We also talked about how or if we want
to work together outside of the LSD Network.
This was a very busy, worthwhile conference,
and one which I was very happy to have
attended. I look forward to next year’s 8th
Annual WORLD Symposium.
- Clinical trials of the oral delivery of
prGCD, using the enzyme replacement therapy
taliglucerase alfa, are planned in the future.
2