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...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2019
Assuntos:
Null Hypothesis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6512882/
https://ncbi.nlm.nih.gov/pubmed/31036580
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000006932
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