Â鶹Éç

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 (Â鶹Éç). Â鶹Éç, 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 (Â鶹Éç) 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…

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 (Â鶹Éç) 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 Â鶹Éç…

Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the progression of Duchenne muscular dystrophy (Â鶹Éç), 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 (Â鶹Éç) 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 (Â鶹Éç) — all seeking to increase production of the dystrophin protein by modulating how the Â鶹Éç gene is read — are now being explored in clinical trials, according to researchers. Updates on several of these investigational Â鶹Éç 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)…