Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults

Abstract Background Enzyme replacement therapy with olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is indicated for non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children and adults. An ongoing, open-label, long-term study (NCT02004704) a...

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Bibliographic Details

Main Authors:	Robin H. Lachmann, George A. Diaz, Melissa P. Wasserstein, Nicole M. Armstrong, Abhimanyu Yarramaneni, Yong Kim, Monica Kumar
Format:	Artigo
Language:	Inglês
Published:	BMC 2023-04-01
Series:	Orphanet Journal of Rare Diseases
Subjects:	Olipudase alfa Recombinant human acid sphingomyelinase Acid sphingomyelinase deficiency Niemann-Pick disease type B and type A/B
Online Access:	https://doi.org/10.1186/s13023-023-02700-x
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