Duchenne Muscular Dystrophy (DMD)

Prosensa Will Pursue Accelerated Approval for Drisapersen to Treat DMD

Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.

DMD: Idebenone Slows Decline of Respiratory Function

Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.

DMD: Drisapersen Dosing Planned for Later in 2014

Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.

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

Pages