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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...

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Foilsithe in:BMC Neurol
Main Authors: Merlini, Luciano, Sabatelli, Patrizia
Formáid: Artigo
Teanga:Inglês
Foilsithe: BioMed Central 2015
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4549867/
https://ncbi.nlm.nih.gov/pubmed/26306629
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12883-015-0408-z
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