Gaucher Disease
Amicus Therapeutics Announces Preliminary Results of Phase 2 Study with Plicera(TM) for Gaucher Disease
Published in Blood Weekly, October 22nd, 2009
Amicus Therapeutics (NASDAQ:FOLD) announced preliminary results from its Phase 2 randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera™ (afegostat tartrate), in treatment-naive adult patients with type 1 Gaucher disease. Two dose regimens of Plicera (225 mg three days on/four days off and seven days on/seven days off) were studied during this six month trial. While all patients enrolled experienced an increase in the level of the target enzyme (GCase) as measured in white blood cells, clinically meaningful improvements in key measures of disease were observed in just one of the eighteen patients who completed the...
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Source: Blood Weekly (2009-10-22)
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