News

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 19, 2017July 19, 2017

Translarna Beneficial for Patients in Transitional Stage of Nonsense Mutation Duchenne MD, Study Finds

News

The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) showed that it can ... Read more

July 18, 2017July 18, 2017

MDA Awards Neurologist Johanna Hamel $130,000 Clinical Research Training Fellowship

News

The Muscular Dystrophy Association (MDA) has awarded Dr. Johanna Hamel, a neurologist at New York’s University of Rochester, a $130,000 fellowship to help her research the underlying mechanisms that cause myotonic dystrophy — the ... Read more

July 17, 2017

Everest to End Duchenne to Hold Second Fundraising Trek to Mount Everest Base Camp

News

Everest to End Duchenne, an organization raising funds to support research to end Duchenne muscular dystrophy, is looking for hikers, outdoor enthusiasts and climbers to take part in its ... 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 12, 2017July 12, 2017

Problems in RNA Processing Seen to Lead to Congenital Myotonic Dystrophy in Early Study

News

The molecular process that leads to congenital myotonic dystrophy (CMD) in infants and children was identified by researchers working with tissue samples and mice. Their study, titled “Disrupted prenatal RNA processing and ... Read more

July 11, 2017

Most Common Congenital Muscular Dystrophy in UK Is MDC1A, 12-year Analysis Shows

News

A comprehensive 12-year genetic and clinical analysis of unrelated patients with congenital muscular dystrophies (CMDs) in the U.K. has confirmed MDC1A as the the most common CMD subtype, and ... Read more

July 10, 2017

Hand Braces During Sleep Can Help Preserve Muscle Function of Boys with Duchenne MD, Study Suggests

News

Overnight use of hand braces preserves hand function and delays the development of shortened muscles or joints in boys who have Duchenne muscular dystrophy (DMD), a study shows. The research, “Evaluation ... 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

July 6, 2017July 6, 2017

Heart Failure Diagnosis and Therapy Reviewed in Duchenne Muscular Dystrophy Patients

News

End-stage heart failure is increasingly becoming the main cause of death in patients with Duchenne muscular dystrophy (DMD). Because cardiomyopathy, an abnormal heart condition, is difficult to detect, a ... Read more