A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results
Melissa Wasserstein(Icahn School of Medicine at Mount Sinai), Monica Kumar(Sanofi (United States)), Michel Tchan(Westmead Hospital), Robin Lachmann(University College London), Laila Arash‐Kaps(Johannes Gutenberg University Mainz), Sandy A. Furey(Sanofi (United States)), Maurizio Scarpa(University of Udine), Carla E. M. Hollak(Amsterdam UMC Location University of Amsterdam), Renata C. Gallagher(Stanford University), Jesús Villarrubia(Hospital Universitario Ramón y Cajal), Olivier Lidove(Groupe Hospitalier Diaconesses Croix Saint-Simon), Eubekir Senates(Istanbul Medeniyet University), Takayuki Ikezoe(Fukushima Medical University), Antonio Barbato(University of Padua), Eugen Mengel(University of Hohenheim), Paulina Mabe(Clinica Santa Maria), Beth L. Thurberg(Sanofi (United States)), Roberto Giugliani(Instituto de Biologia Molecular e Celular), Norberto Guelbert, Yixin Chen(Nanjing Drum Tower Hospital), Atef Zaher(Sanofi (United States))
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