A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy

OBJECTIVE: To investigate the efficacy and safety of aceneuramic acid extended-release (Ace-ER), a treatment intended to replace deficient sialic acid, in patients with GNE myopathy. METHODS: UX001-CL301 was a phase 3, double-blind, placebo-controlled, randomized, international study evaluating the...

Descripción completa

Detalles Bibliográficos
Autores principales: Lochmüller, Hanns, Behin, Anthony, Caraco, Yoseph, Lau, Heather, Mirabella, Massimiliano, Tournev, Ivailo, Tarnopolsky, Mark, Pogoryelova, Oksana, Woods, Catherine, Lai, Alexander, Shah, Jinay, Koutsoukos, Tony, Skrinar, Alison, Mansbach, Hank, Kakkis, Emil, Mozaffar, Tahseen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2019
Materias:
Null Hypothesis
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6512882/
https://www.ncbi.nlm.nih.gov/pubmed/31036580
http://dx.doi.org/10.1212/WNL.0000000000006932

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6512882/
https://www.ncbi.nlm.nih.gov/pubmed/31036580
http://dx.doi.org/10.1212/WNL.0000000000006932

Ejemplares similares