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

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

Published:2022-07 Issue:7 Volume:24 Page:1425-1436
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

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

Funder

Sanofi Genzyme

Sanofi US

Publisher

Elsevier BV

Subject

Genetics (clinical)

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