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UK Phase 2/3 acid sphingomyelinase deficiency trial begins

Sanofi Genzyme has enrolled and dosed the first adult patient in the UK in a Phase 2/3 clinical trial for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B.




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