News

September 20, 2017September 20, 2017

Phrixus, DMD Groups Plan First Clinical Trial of Potential Duchenne Therapy Already Used in Europe

Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no ... Read more

September 18, 2017September 18, 2017

‘Coach to Cure MD’ and Other Fundraising Events Mark Duchenne Muscular Dystrophy Month

News

For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month by using college football games to raise awareness of Duchenne muscular ... Read more

September 15, 2017

Santhera to Appeal European Agency’s Decision Against Raxone as a Duchenne MD Therapy

News

The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne muscular dystrophy (DMD) treatment. Santhera said in a press release that it plans to ... Read more

September 14, 2017September 16, 2017

Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy

News

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). ... Read more

September 13, 2017September 13, 2017

Shortened Telomeres Linked to Muscle Degeneration in Duchenne, Study Finds

News

Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able to build new muscle, according to new research. A full ... Read more

September 12, 2017

Utah Researcher Receives Grant to Study Stages of Congenital Myotonic Dystrophy

News

A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas ... Read more

September 12, 2017September 19, 2017

First DMD Patients to Receive Raxone Under U.K.’s Early Access Program

News

The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to ... Read more

September 11, 2017September 11, 2017

Sarepta’s Exon 53 Skipping Therapy, Golodirsen, Improves Dystrophin Expression in Phase 1/2 Trial

News

Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in a first clinical trial of DMD patients. According to results of ... Read more

September 8, 2017

On World Duchenne Awareness Day, Winners of 2017 STRIVE Awards Announced

News

PTC Therapeutics recently announced the recipients of the 2017 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) Awards program, recognizing excellence and innovation in the Duchenne muscular dystrophy (DMD) community. ... Read more

September 7, 2017

Parent Project Muscular Dystrophy Holding Duchenne Action Month During September

News

Parent Project Muscular Dystrophy is holding an awareness, fund-raising and educational campaign throughout September to fight Duchenne muscular dystrophy (DMD). It has scheduled events every day of Duchenne Action Month. You’ll ... Read more