Marisa Wexler, MS, senior science writer —

Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and breathing ability before they lose the ability to walk, a new study reports. The findings have major implications for clinical trials testing treatments that aim to boost arm and/or lung function in DMD. Traditionally,…

The U.S. Food and Drug Administration (FDA) has given the green light for Myogenica to start a clinical trial testing its stem cell therapy MyoPAXon in people with Duchenne muscular dystrophy (DMD). DMD, the most common type of muscular dystrophy, is caused by mutations in the…

Satellos Bioscience is gearing up to launch a Phase 1 clinical trial of its Duchenne muscular dystrophy (DMD) treatment SAT-3247. The company said it has submitted an application to authorities in Australia seeking permission to start the trial, which is expected to test the safety and pharmacological properties…

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…

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…

PTC Therapeutics plans to soon ask the U.S. Food and Drug Administration (FDA) to approve ataluren as a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. “We are thrilled to announce that we are moving forward with the submission of a new drug application for…

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,…