GENZYME TO OFFER ACCESS
TO EXPERIMENTAL POMPE’S TREATMENT
TUCSON, Ariz., Sept. 15, 2003 — Biotechnology firm Genzyme of
Cambridge, Mass., will offer an access program for its experimental
treatment for Pompe’s
disease once all regulatory matters have been settled. The program
will focus on severely ill patients who are unable to participate in
clinical studies.
The experimental enzyme, which Genzyme calls Myozyme, appears to compensate
for a lack of the natural enzyme acid maltase (also known as acid alpha-gluocosidase)
in the cells of those with acid maltase deficiency (Pompe’s disease).
Acid maltase normally breaks down stored sugar.
Genzyme, which is now sponsoring clinical trials of this enzyme for infants and for children
under 3, also plans to launch a Web-based registry that will collect
data about the course, outcomes and management of Pompe’s disease,
and a small study of the natural course of late-onset Pompe’s.
The natural history data will likely be used for a Myozyme trial in
2004.
MDA-supported laboratory research helped lay the foundation for the
development of Myozyme, and MDA is partially supporting the Myozyme
trials.
For information about Pompe’s disease studies and the access
program, contact Genzyme’s Medical Information Department at medinfo@genzyme.com or (800) 745-4447.
In Europe, e-mail eumedinfo@genzyme.com,
or call 31-35-699-1499.
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