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Genzyme Presents Data from its Phase 1b Program for Niemann-Pick Type B at the Lysosomal Disease Network’s WORLD Symposium 2015. Study showed tolerability of dose-escalation regimen.

CAMBRIDGE, Mass. --Genzyme, a Sanofi company, today presented data from its Phase 1b clinical study at the Lysosomal Disease Network’s WORLD Symposium 2015 in Orlando, Fla. detailing the investigational use of enzyme replacement therapy in the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type B), a lysosomal storage disease caused by genetic mutations that affect the metabolism of sphingomyelin. The Genzyme study evaluated the tolerability and safety of olipudase alfa (recombinant human acid sphingomyelinase) in five adult patients with ASMD.
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Genzyme Presents Phase 3 Clinical Trial Extension Results for Cerdelga® (eliglustat) at Lysosomal Disease Network’s WORLD Symposium 2015

CAMBRIDGE, Mass. -- Genzyme, a Sanofi company, today reported extension study data from its Phase 3 ENGAGE and ENCORE studies of Cerdelga® (eliglustat), a first-line oral therapy approved by the FDA and the European Commission for the treatment of certain adults with Gaucher disease type 1. The results from the studies were presented today at the 11th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Fla. Both extension studies demonstrated continued stability and/or improvements across established end points and published therapeutic treatment goals.
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