Patricia Inácio, PhD, science writer —

The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and age at symptom onset, according to the findings of a four-year study, but the researchers nonetheless reported “notable variability” with patients’ general decline. The study, by researchers in the Netherlands, focused on changes over…

Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD), according to top-line data from the REACH Phase 3 trial. Participants given losmapimod did see improvements over 48…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage…

Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our…

Patients and experts in limb-girdle muscular dystrophy (LGMD), along with drug developers, community leaders, and regulatory agency personnel, came together for the LGMD Scientific Workshop to discuss how to tackle the unmeet therapeutic needs of people with these muscle-wasting diseases. The workshop, held in early February in Maryland,…

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead treatment candidate for myotonic dystrophy type 1 (DM1). Orphan drug status is designed to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…

Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E (LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without…

Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older. This approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows a similar decision in the European Union. In the U.S., Agamree is approved for patients…

Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely seen only in adults — and type of mutation, a three-year study of BMD’s natural history shows. “We … provide evidence that decline/improvement in function would be hard to measure…

The HOPE-3 clinical trial testing Capricor Therapeutics’ experimental cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD) can continue as planned, the company announced. That’s the result of an interim futility analysis, which evaluates whether it is mathematically possible for a trial to achieve its goal — if not, the trial…