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

Background: GNE Myopathy (GNEM) is a progressive adult-onset myopathy likely caused by deficiency of sialic acid (SA) biosynthesis. Objective: Evaluate the safety and efficacy of SA (delivered by aceneuramic acid extended-release [Ace-ER]) as treatment for GNEM. Methods: A Phase 2, randomized, doubl...

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Detalles Bibliográficos
Autores principales: Argov, Zohar, Caraco, Yoseph, Lau, Heather, Pestronk, Alan, Shieh, Perry B., Skrinar, Alison, Koutsoukos, Tony, Ahmed, Ruhi, Martinisi, Julia, Kakkis, Emil
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2016
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271423/
https://www.ncbi.nlm.nih.gov/pubmed/27854209
http://dx.doi.org/10.3233/JND-159900

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271423/
https://www.ncbi.nlm.nih.gov/pubmed/27854209
http://dx.doi.org/10.3233/JND-159900

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