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Recent highlights in Neuromuscular Research

Ataluren Clinical Data Demonstrate an Increase in Dystrophin Expression in Duchenne Muscular Dystrophy Patients

- Data Published in PLOS ONE -

South Plainfield, NJ – December 12, 2013 – PTC Therapeutics, Inc.,(NASDAQ: PTCT) today announced the publication of data in PLOS ONE demonstrating that nonsense mutation Duchenne muscular dystrophy (nmDMD) patients treated with ataluren, an investigational new drug, experienced an increase in dystrophin expression. These data were obtained from PTC’s Phase 2a open-label trial of ataluren in which change in full-length dystrophin expression, as assessed by immunofluorescent staining, was the primary endpoint. Read more

Research conducted by Michael Rudnicki at Ottawa Hospital Research Institute reveals that injecting the protein Wnt7a into mice with Duchenne muscular dystrophy increased muscle strength almost two-fold – to nearly normal levels. They also found that the size of the mice muscle fibre increased and there was less muscle damage, compared to mice not given Wnt7a. Read more.

GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen in patients with Duchenne muscular dystrophy. Read press release.

Toshifumi Yokota, Friends of Garrett Cumming Research Chair at the University of Alberta, discovered a mutation that causes a significant increase in dystrophin protein in mice. “If we can find the mechanism that causes the dystrophin protein to regrow, it would be a drug target for the treatment of Duchenne muscular dystrophy,” said Yokota. “Our discovery is very promising.” Watch a news story

What The Delay Of A Promising Muscular Dystrophy Drug Means For Patients, Investors And All Of Biotech. Written by Matthew Herper, Published online. November 12, 2013.

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