Steve Bryson, PhD,  science writer—

Data from two clinical trials confirm that Agamree (vamorolone), an approved anti-inflammatory treatment for people with Duchenne muscular dystrophy (DMD), blocks the action of a receptor involved in regulating the balance of salt and water in the body, and may have advantages over traditional corticosteroids. A study found…

The first patient has been dosed as part of a Phase 1b clinical trial of AAV1.SERCA2 (SRD-003), Sardocor‘s one-time gene therapy for cardiomyopathy — a type of heart disease — associated with Duchenne muscular dystrophy (DMD-CM). The multicenter trial, dubbed MUSIC-DMD (NCT06224660), is being conducted in collaboration…

Nearly one-third of people with muscular dystrophy (MD) experience moderate pain, which varies by the type of their MD, a study with more than 1,200 participants suggests. To manage their pain, about a fifth of patients used prescribed pain medications, mostly non-opioid therapies at first, then combined with opioids…

Households incur substantial costs to accommodate people with Duchenne muscular dystrophy (DMD), according to a survey of patient caregivers in the U.S. Costs, such as those for purchasing and/or modifying an accessible vehicle, relocating to or building an accessible home, modifying home entrances, and buying medical equipment, were particularly…

Researchers identified a unique protein signature in the bloodstreams of adults with Becker muscular dystrophy (BMD) and limb-girdle muscular dystrophy (LGMD) that significantly differs from healthy individuals after exercise. “We propose that a subset of circulating proteins may be more indicative of disease progression and/or therapeutic efficacy than…

The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare…

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the therapy’s developer, Sarepta Therapeutics, said it is continuing to gather and analyze information about this event, which…

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the U.S.-based Muscular Dystrophy Surveillance, Tracking and Research Network, known as MD STARnet. However, despite finding such benefits with prophylactic,…

Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global clinical trial able to walk. That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which…

The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new treatment to, among other goals, slow or stop the progression of the muscle-wasting disease. Formed…