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(Unknown), Carla E. M. Hollak(Centre For Development Studies), 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(University of California, Los Angeles), Antonio Barbato(Federico II University Hospital), Eugen Mengel(University of Hohenheim), Paulina Mabe(Clinica Santa Maria), Beth L. Thurberg(Sanofi (United States)), Roberto Giugliani(Universidade Federal do Rio Grande do Sul), Norberto Guelbert, Yixin Chen(Nanjing Drum Tower Hospital), Atef Zaher(Sanofi (United States))
Cited by 99
Related Papers
Elevated globotriaosylsphingosine is a hallmark of Fabry disease
|Proceedings of the National Academy of Sciences|2008|725
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
|New England Journal of Medicine|2016|555
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
|European Journal of Pediatrics|2007|522
Primary ciliary dyskinesia: a consensus statement on diagnostic and treatment approaches in children
|European Respiratory Journal|2009|520