Olipudase alfa approved for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

Bibliographic Details
Main Authors:	Monica Penon-Portmann, Sheri A. Poskanzer, Jaya Ganesh, Irene Chang
Format:	Article
Language:	English
Published:	Elsevier 2023-01-01
Series:	Genetics in Medicine Open
Subjects:	Acid sphingomyelinase deficiency Enzyme replacement therapy Lysosomal storage disease Niemann-Pick disease Olipudase alfa
Online Access:	http://www.sciencedirect.com/science/article/pii/S2949774423007896

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