Marisa Wexler, MS,  senior science writer—

Note: This story was updated Oct. 22, 2024, to remove a reference to the therapy being pulled from the market in Europe. Although this was the initial recommendation, the therapy has remained available in Europe.  PTC Therapeutics plans to soon ask the U.S. Food and Drug Administration (FDA)…

Connecting with a supportive community is essential for people affected by muscular dystrophy to maintain both their emotional and physical well-being, according to Brandon Kozar, a psychologist at Nationwide Children’s Hospital. “When we come together … something amazing happens. We become more powerful. We become connected. At the very least,…

Parent Project Muscular Dystrophy (PPMD) held its first conference in 1994, in Orlando, Florida. A year later, at its meeting in Pittsburgh, fewer than two dozen people were in attendance, just a small group of parents of children with Duchenne muscular dystrophy (DMD) and a few scientists interested…

Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application seeking approval of the company’s CAP-1002 cell therapy, now named deramiocel, as a treatment for Duchenne muscular dystrophy (DMD). The FDA has scheduled the pre-biologics license application (BLA)…

The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) the chance to “celebrate three decades of progress, community, and resilience.” The PPMD conference, which runs through Saturday, is taking place…

The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene therapy for individuals with Duchenne muscular dystrophy (DMD) ages 4 and older regardless of their ability to walk. Previously, the treatment had been approved for DMD children ages 4…

Duchenne muscular dystrophy (DMD) can make it harder to engage in social activities, and patients often experience grief and frustration over lost function. Family, however, can be an invaluable source of support to help overcome the challenges of the disease. That’s according to a study, “‘You…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…

Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports. Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which…

Treatment with an antioxidant supplement led to improvements in muscle strength and quality of life for people with facioscapulohumeral muscular dystrophy (FSHD) in a small clinical trial. The use of such supplements was particularly seen to improve the muscle quality of patients’ quadriceps — a group of muscles found…