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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