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Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype

2 Sept 2015

Abstract excerpt

BACKGROUND: Exon skipping therapy is an emerging approach in Duchenne Muscular Dystrophy (DMD). Antisense oligonucleotides that induce skipping of exon 51, 44, 45, or 53 are currently being evaluated in clinical trials. These trials were designed on the basis of data available in general DMD population. OBJECTIVES: Our objective was to compare the clinical and functional statuses of non-ambulant DMD patients...

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PMID: 27858743
DOI: 10.3233/jnd-150100

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