Duchenne Muscular Dystrophy (DMD)

UPDATE: DMD: Encouraging Results for SMT C1100

 

DMD: Ataluren May Get Conditional Approval in Europe

The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.

DMD: Sarepta Will Pursue Accelerated Approval for Eteplirsen and Conduct Additional Trials

Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S.

Tadalafil, Sildenafil Increase Blood Flow to DMD-Affected Muscles

A study conducted in 10 boys with Duchenne muscular dystrophy (DMD) has found that blood flow to exercising muscles is deficient and that treatment with either tadalafil (Cialis) or sildenafil (Viagra) normalizes this blood flow, at least in the short term (after one dose of either drug).

Tadalafil and sildenafil, both PDE5 inhibitors, are approved by the U.S. Food and Drug Administration (FDA) to treat erectile dysfunction.

A Landmark Development for the Duchenne MD Community

DMD: Prosensa Pursuing Path Forward for Drisapersen

Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.

MDA Thrilled FDA Will Consider Accelerated Approval for New Muscle Disease Drug Aimed at DMD

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

Shooting for Hope

Artist: 

Bryson is an honor roll student at school. His favorite subjects are reading, spelling and social studies, but sports are his true passion. He collects sports memorabilia. Bryson served two years as MDA’s National Goodwill Ambassador from 2011 – 2013. He and his parents traveled throughout the United States attending events, meeting with sponsors and spreading the message of MDA’s mission. He appeared on the Labor Day Telethon as a co-announcer for two years.

DMD: Phase 2 Drisapersen Results Encouraging

Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014.

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