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Article

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

Journal of neuromuscular diseases - 1 Jan 2021

McDonald Craig M, Shieh Perry B, Abdel-Hamid Hoda Z, Connolly Anne M, Ciafaloni Emma, Wagner Kathryn R, Goemans Nathalie, Mercuri Eugenio, Khan Navid, Koenig Erica, Malhotra Jyoti, Zhang Wenfei, Han Baoguang, Mendell Jerry R

Abstract excerpt

BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production.ObjectiveTo report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficacy and safety of eteplirsen in a larger cohort.MethodsAmbulatory patients aged 7-16 years, with confirmed mutations...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 34120909
DOI: 10.3233/JND-210643
PMCID: PMC8673535

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