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Treatment with the gene therapy BB-301 led to improved swallowing for the first person with oculopharyngeal muscular dystrophy (OPMD) to receive the treatment as part of an ongoing clinical trial, according to new interim data from BB-301’s developer Benitec Biopharma. “We are pleased to report continued positive…

One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults with facioscapulohumeral muscular dystrophy (FSHD), according to an open-label pilot study. Blood and muscle tests also showed the therapy candidate successfully engaged with its intended target and had a…

A pair of identical twins with myotonic dystrophy type 1 (DM1) were found to have different deficits in social cognition that corresponded with differing patterns of brain tissue loss on MRI scans, a case report shows. Researchers believe the findings highlight that, while genetic factors can contribute to brain development,…

With the first gene therapy for Duchenne muscular dystrophy (DMD) now approved in the U.S. for most people with the disease, clinical trials are underway to better understand the safety and efficacy of the approved treatment, find strategies to expand its use, and develop the next generation of DMD…

Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the progression of Duchenne muscular dystrophy (DMD), were spotlighted in a session at last week’s Parent Project Muscular Dystrophy (PPMD) conference. In Duchenne MD, mutations lead to a…

In its 30-year history, Parent Project Muscular Dystrophy (PPMD) has helped fund the development of many experimental treatments for Duchenne muscular dystrophy (DMD) and some of these are about to be tested in clinical trials. Several of these up and coming treatments, including strategies for gene…

More than half a dozen exon-skipping therapies for Duchenne muscular dystrophy (DMD) — all seeking to increase production of the dystrophin protein by modulating how the DMD gene is read — are now being explored in clinical trials, according to researchers. Updates on several of these investigational DMD treatments…

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…