Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months

Olipudase alfa, a recombinant human acid sphingomyelinase (ASM), is an enzyme replacement therapy for the treatment of nonneurologic manifestations of acid sphingomyelinase deficiency (ASMD). This ongoing, open-label, long-term study (NCT02004704) assessed safety and efficacy of olipudase alfa follo...

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Bibliographic Details
Published in:J Inherit Metab Dis
Main Authors: Wasserstein, Melissa P., Diaz, George A., Lachmann, Robin H., Jouvin, Marie-Hélène, Nandy, Indrani, Ji, Allena J., Puga, Ana Cristina
Format: Artigo
Language:Inglês
Published: Springer Netherlands 2018
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6133173/
https://ncbi.nlm.nih.gov/pubmed/29305734
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-017-0123-6
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