News

People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but are encouraged to transition their care to telemedicine and to medical tests performed at home to minimize risk. Those are among the consensus recommendations provided by neuromuscular specialists to help these patients…

CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ potential non-viral gene therapy for people with Duchenne muscular dystrophy (DMD). The money is expected to advance a platform that delivers the full DMD gene to the muscles…

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar hosted by Parent Project Muscular Dystrophy (PPMD). PTC is the maker of Emflaza…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders. The team consists of three physicians, all experts in these conditions and leading clinicians at an…

Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular dystrophy (DMD) in a Phase 3 trial of its investigational treatment givinostat, the company said in a recent webinar hosted by Parent Project Muscular Dystrophy. Although treatment of current…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Traci and Tony Rico of Cardiff-by-the-Sea, California, juggle more than their share of family health issues. Their son, Tanner, has Duchenne muscular dystrophy, and their daughter, Pria, has recurrent respiratory papillomatosis (RPP) — a rare genetic condition that has led to 97 surgeries to remove tumors in her larynx…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s EB 002 ((+)-epicatechin) for the treatment of Duchenne and Becker muscular dystrophies. The designation supports the development of experimental treatments for rare diseases (those that affect fewer than 200,000 people in the…