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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Detalles Bibliográficos
Autores principales: Wasserstein, Melissa P., Diaz, George A., Lachmann, Robin H., Jouvin, Marie-Hélène, Nandy, Indrani, Ji, Allena J., Puga, Ana Cristina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6133173/
https://www.ncbi.nlm.nih.gov/pubmed/29305734
http://dx.doi.org/10.1007/s10545-017-0123-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6133173/
https://www.ncbi.nlm.nih.gov/pubmed/29305734
http://dx.doi.org/10.1007/s10545-017-0123-6

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