Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.

Nuria Carrillo-CarrascoMay C MalicdanPetcharat LeoyklangJoseph A ShraderGalen JoeChristina SlotaJohn PerreaultJohn D HeissBradley ClassChia-Ying LiuKennan BradleyColleen JodarskiCarla CicconeClaire DriscollRebecca ParksScott Van WartLevent BaymanChristopher S CoffeyMelanie QuintanaScott M BerryMarjan HuizingWilliam A Gahl

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2021)

ManNAc showed long-term safety, biochemical efficacy consistent with the intended mechanism of action, and preliminary evidence clinical efficacy in patients with GNE myopathy.

Keyphrases

late onset
muscular dystrophy
open label

early onset
clinical trial
double blind

randomized controlled trial
placebo controlled
myasthenia gravis

study protocol
duchenne muscular dystrophy