Improving clinical trial design for Duchenne muscular dystrophy

Currently, the most promising therapies for Duchenne muscular dystrophy (DMD) are exon skipping and stop codon read-through, two strategies aimed at restoring the expression of dystrophin. A phase 3 clinical trial with drisapersen, a drug designed to induce exon 51-skipping, has failed to show signi...

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Veröffentlicht in:	BMC neurology 2015-08, Vol.15 (1), p.153-153, Article 153
Hauptverfasser:	Merlini, Luciano, Sabatelli, Patrizia
Format:	Artikel
Sprache:	eng
Schlagworte:	Adrenal Cortex Hormones - therapeutic use Clinical Trials as Topic - methods Codon Debate Disease Progression Drug Therapy, Combination Dystrophin - genetics Exons Humans Male Muscle Strength Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - physiopathology Oligonucleotides - therapeutic use Quality Improvement Research Design Treatment Outcome Walking
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