Research News

Prosensa announces commencement of re-dosing of drisapersen in North America in patients with Duchenne muscular dystrophy

Leiden, The Netherlands, Sept. 17, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that a comprehensive program of re-dosing has commenced, with the first patients now re-dosed in the United States. All dosing in the drisapersen clinical program had been placed on hold by GSK on September 20, 2013, upon announcement of the DEMAND III study results.

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Development of Neuromuscular Network to enhance research and clinical care

June 12, 2014 – Muscular Dystrophy Canada is thrilled to announce the creation of a pan-Canadian neuromuscular disease network. The network, which will be co-funded by CIHR and Muscular Dystrophy Canada, will involve researchers and neuromuscular disease clinical care personnel from across the country. It will empower patients and families through improved access to needed information, resources, and connections with each other through a patient portal. READ MORE

Neuromuscular Respiratory Care Research Grant Funding Opportunity Launched

Muscular Dystrophy Canada has launched a request for proposals aimed at addressing gaps in knowledge and enhancing knowledge translation related to respiratory care. Improving the respiratory health of people affected by neuromuscular disorders is a strategic priority for Muscular Dystrophy Canada. We are committed to funding research to advance the respiratory health, health care services, and quality of life for people living with neuromuscular disorders. Read more

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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