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Genzyme,
a Sanofi company, today announced the publication of results from the
ENGAGE registration study evaluating Cerdelga® (eliglustat) in
treatment-naïve patients with Gaucher disease type 1 in the February 17,
2015 issue
of The Journal of the American Medical Association.
The ENGAGE study is a Phase 3 randomized, double-blinded,
placebo-controlled, multinational registration trial of 40 eligible
treatment-naïve patients with Gaucher disease type 1 who had
splenomegaly in addition to thrombocytopenia and/or anemia at study
entry. Gaucher disease is a genetic disorder in which individuals fail
to produce glucocerebrosidase, an important enzyme that breaks down the
glycolipid glucocerebroside within lysosomes. In the absence of
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