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A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results

Research output: Contribution to journal › Article › Academic › peer-review

DOI

https://doi.org/10.1016/j.gim.2022.03.021
Final published version

Melissa Wasserstein
Robin Lachmann
Laila Arash-Kaps
Antonio Barbato
Renata C. Gallagher
Roberto Giugliani
Norberto Bernardo Guelbert
Takayuki Ikezoe
Olivier Lidove
Paulina Mabe
Eugen Mengel
Maurizio Scarpa
Eubekir Senates
Michel Tchan
Jesus Villarrubia
Yixin Chen
Sandy Furey
Beth L. Thurberg
Atef Zaher
Monica Kumar

Original language	English
Pages (from-to)	1425-1436
Number of pages	12
Journal	Genetics in medicine
Volume	24
Issue number	7
DOIs	https://doi.org/10.1016/j.gim.2022.03.021
Publication status	Published - 1 Jul 2022

Research areas

Diffusing capacity of the lung for carbon monoxide, Niemann-Pick type A/B, Niemann-Pick type B, Organomegaly, Recombinant human acid sphingo-myelinase

ID: 25205900