DMD

September 15, 2017

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

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 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

August 2, 2017August 2, 2017

New Gene Therapy to Fix Dystrophin Deficiency in DMD Shows Promise in Mice, Study Shows

News

Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene to muscles as a way to correct the faulty ... Read more

July 28, 2017July 28, 2017

Microdystrophin Gene Therapy Shows Promise in Dogs with Duchenne Muscular Dystrophy, Study Shows

News

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, ... Read more

July 20, 2017

Sarepta Launches Exondys 51 Managed Access Program in Europe, North and South America

News

Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne ... Read more

July 14, 2017

FDA Grants Orphan Designation to Mallinckrodt’s MNK-1411 for DMD

News

The U.S. Food and Drug Administation has granted orphan designation to Mallinckrodt Pharmaceuticals‘ drug candidate MNK-1411 for treatment of Duchenne Muscular Dystrophy (DMD). MNK-1411 is an injection composed of a formulation ... Read more

July 7, 2017July 7, 2017

Experts Review Current and Potential Treatments for Duchenne Muscular Dystrophy

News

Experts provided an overview of the many approaches to counteract the loss of muscle mass, inflammation, fibrosis, calcium overload, oxidative stress, and ischemia (inadequate blood supply) in Duchenne muscular ... Read more

June 26, 2017June 26, 2017

Edasalonexent Could Reduce Functional Decline in Boys with DMD, MoveDMD Phase 2 Results Suggest

News

Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent ... Read more

June 12, 2017June 12, 2017

Sarepta Recruits Duchenne Boys for Phase 3 ESSENCE Trial to Test 2 Potential DMD Therapies

Duchenne Muscular Dystrophy News, News

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is ... Read more

May 19, 2017

CAP-1002 Positive Results Support New Clinical Trial

News

Positive results from the HOPE-Duchenne clinical trial support further clinical development of Capricor Therapeutics’ therapy candidate CAP-1002 for treatment of cardiac anomalies caused by Duchenne Muscular Dystrophy (DMD). The company ... Read more