Forest Ray PhD,  —

Losmapimod, a potential oral treatment for facioscapulohumeral muscular dystrophy (FSHD), missed its main goal in a recent Phase 2 clinical trial, but its developer, Fulcrum Therapeutics, sees this as a constructive failure. Lessons learned will help to shape losmapimod’s continued development, which is supported by the secondary…

AMRA Medical’s new MRI method was found to play an instrumental role in detecting changes in disease progression among individuals with facioscapulohumeral muscular dystrophy (FSHD) in a recently completed clinical trial, according to the company. The new MRI technique, introduced in the Phase 2b ReDUX4 study, provides…

Givinostat halted muscle shrinkage and fat infiltration in men with Becker muscular dystrophy (BMD) despite failing to ease muscle scarring, compared to a placebo, according to top-line, one-year results of an ongoing Phase 2 clinical trial. “We are very encouraged by the significant difference in muscle fat infiltration…

FibroGen has begun a second Phase 3 clinical trial of pamrevlumab, given with corticosteroids, among boys with Duchenne muscular dystrophy (DMD) who are able to walk. The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas…

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Treatment with Amondys 45 was safe and well-tolerated, showing little to no evidence of accumulation in the blood…

Treatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability to walk — in boys with Duchenne muscular dystrophy (DMD), new data from a long-term Phase 2/3 clinical study show. The new data, from an ongoing seven-year study, was presented by Paolo Bettica,…

In the first year since Parent Project Muscular Dystrophy (PPMD) and Thread launched a mobile app aimed at making participation easier, engagement in the Duchenne Registry — a newly revamped, 12-year-old network of patient-powered data — increased more rapidly than ever before, the non-profit reported. From November…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the first Phase 3 DMD gene therapy program to begin enrolling eligible participants, is an important milestone for the…

The investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys with Duchenne muscular dystrophy (DMD) — and to improvements in their motor abilities, according to top-line results of a Phase 2 trial. However, following almost one year of the Sarepta…