Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study.

Argov, Zohar; Caraco, Yoseph; Lau, Heather; Pestronk, Alan; Shieh, Perry B; Skrinar, Alison; Koutsoukos, Tony; Ahmed, Ruhi; Martinisi, Julia; Kakkis, Emil.
J Neuromuscul Dis; 3(1): 49-66, 2016 03 03.
Artigo em Inglês | MEDLINE | ID: mdl-27854209