Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
An experimental drug designed to reduce scarring in Duchenne MD-affected muscles shows signs of safety and desired activity; FDA will speed its review
HT-100, an experimental drug being developed for Duchenne muscular dystrophy (DMD) with support from MDA, has shown preliminary safety and signs of the desired effects on scar tissue formation in the first 17 trial participants, and it has received "fast track" designation from the U.S. Food and Drug Administration (FDA).
A trial of an experimental compound to counteract muscle inflammation and scarring in Duchenne MD resumes, after an interruption to complete a dog safety study
Update (July 3, 2014): Akashi says the HT-100 main trial is now closed; the extension study is open to those who have already participated in the main trial.
MDA has responded to questions from the US House of Representatives about how to improve and speed up the drug development process
Last month, the Energy & Commerce Committee of the U.S. House of Representatives launched a bipartisan effort called the 21st Century Cures initiative to accelerate the process by which drugs are discovered and delivered to patients. Congress has sought input from the patient community and advocacy organizations like MDA.
Isis Pharmaceuticals is launching a phase 1 trial of ISIS-DMPKRx, an experimental drug for type 1 myotonic muscular dystrophy
Update (June 11, 2014): This story has been updated with additional information about the phase 1 trial of ISIS-DMPKRx in healthy volunteers and the planned trial in people with type 1 MMD, as well as the availability of a fact sheet on this drug provided by Isis Pharmaceuticals...
The company plans to submit a New Drug Application to the U.S. Food and Drug Administration later this year, and has committed to conducting two confirmatory post-approval clinical trials
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.