News

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…

Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss heart complications associated with Duchenne muscular dystrophy (DMD) and will also provide an update on the development of CAP-1002, its lead cell therapy candidate for people with DMD. The event, at 1:30…

A CureDuchenne Ventures equity investment will support Dyne Therapeutics in developing precision treatments aimed at repairing muscles in Duchenne and other muscular dystrophies. The funding — whose amount was not disclosed — will help to advance the company’s efforts to target the genetic makeup of Duchenne muscular dystrophy (DMD), with a goal…

Invitae and the Muscular Dystrophy Association (MDA) are partnering to expand access to free genetic testing and post-test counseling in the United States and Canada, with a goal of more quickly diagnosing muscular dystrophy. Testing is offered at no charge through Invitae’s Detect Muscular Dystrophy program, and is…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. The 30-minute session can be accessed on the MDA’s Facebook page, and starts at 6 p.m. EST. People with…

Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as it should, making it unlikely to raise a person’s COVID-19 risk, Catabasis Pharmaceuticals said. COVID-19 is believed to pose the greatest threat to older people and those…

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a treatment for facioscapulohumeral muscular dystrophy (FSHD). The data, “Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability and Target Engagement,” were presented by Michelle…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the COVID-19 pandemic. Also, the company continues to work with regulatory authorities to guarantee the continuation…