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Improving clinical trial design for Duchenne muscular dystrophy
BACKGROUND: 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 t...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2015
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4549867/ https://www.ncbi.nlm.nih.gov/pubmed/26306629 http://dx.doi.org/10.1186/s12883-015-0408-z |