Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

OBJECTIVE: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. METHODS: Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly gol...

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Publicat a:Neurology
Autors principals: Frank, Diane E., Schnell, Frederick J., Akana, Cody, El-Husayni, Saleh H., Desjardins, Cody A., Morgan, Jennifer, Charleston, Jay S., Sardone, Valentina, Domingos, Joana, Dickson, George, Straub, Volker, Guglieri, Michela, Mercuri, Eugenio, Servais, Laurent, Muntoni, Francesco
Format: Artigo
Idioma:Inglês
Publicat: Lippincott Williams & Wilkins 2020
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7357297/
https://ncbi.nlm.nih.gov/pubmed/32139505
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000009233
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