making muscle wasting history

Leiden University Medical Center and Prosensa B.V. Announce New England Journal of Medicine Publication of First Successful Clinical Study with RNA-based Therapeutic PRO051 in Duchenne Muscular Dystrophy

• Last Edited: 6 Jul 2009
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Novel RNA-based therapeutic PRO051 to enter Phase I/II clinical trials in DMDLeiden, the Netherlands, December 27, 2007. Leiden University Medical Center (LUMC) and Biotech Company Prosensa announ...

Tags: prosensa, exon skipping, clinical trial.

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.