making muscle wasting history

Exon skipping for Duchenne Muscular Dystrophy: a successful clinical trial in the UK

• Last Edited: 5 May 2009
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By Professor Francesco Muntoni, UCL Institute of Child Health LondonProfessor Francesco Muntoni of UCL and Head of the MDEX Consortium discusses the results of the latest successful experimental t...

Tags: Exon skipping, AVI BioPharma, AVI4658, antisense oligonucleotide, exon 51.

2-'O-methyl antisense oligos (PS) targeting exon 51.

• Last Edited: 15 Sep 2010
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BACKGROUND:Duchenne’s muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years. By inducing specific exon sk...

Tags: myofibers, biopsy, exon skipping, exon 51, prosensa, GSK, clinical trials.