News

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on PGN-EDODM1, a medicine being developed by PepGen for myotonic dystrophy type 1, or DM1 — a subtype of the most common form of muscular dystrophy. With the hold lifted, PepGen is ready to…

A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), now will be reviewed by the European…

People with myotonic dystrophy type 1 (DM1) reported gains in measures of muscle strength after being treated with AOC 1001 in a clinical trial. That’s according to new data announced by Avidity Biosciences, the developer of AOC 1001. The findings “reinforce our belief in the potential of AOC 1001…

The gene therapy RGX-202 at lower dose was well tolerated by three boys with Duchenne muscular dystrophy (DMD) in an early clinical trial, and biomarker data suggest the treatment is working as intended. Regenxbio, the company developing RGX-202, expects to administer a higher dose of…

Researchers have created a three-dimensional (3D) artificial muscle model, derived from patient cells, that accurately replicates the damage observed in people with Duchenne muscular dystrophy (DMD). This new model, the scientists believe, will enable more efficient testing of treatments that could potentially reverse such muscle damage. The findings were…

Capricor Therapeutics intends to ask the U.S. Food and Drug Administration (FDA) to approve its experimental cell therapy CAP-1002 for treating Duchenne muscular dystrophy (DMD) in 2025. The company recently completed a meeting with the agency, which confirmed that data from the ongoing Phase 3 clinical…

A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is expanding, with plans to enroll 120 additional adults with the genetic disorder. According to EDG-5506’s developer Edgewise Therapeutics, enrollment in the expanded study is ongoing. The company is hosting…

AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle health in a mouse model of Duchenne muscular dystrophy (DMD). According to AMO Pharma, the treatment’s developer, AMO-02 also improved the animals’ metabolism and cognitive abilities. “These studies…

Covering topics such as mobility equipment and the role of physical therapy, a new, free, online CureDuchenne course seeks to provide guidance and support to Duchenne muscular dystrophy (DMD) caregivers. Specifically, the virtual CureDuchenne Caregiver Course seeks to help caregivers of those newly diagnosed with DMD —…

The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in place for nearly a decade — should now not be renewed, a committee of the European Medicines Agency (EMA) has recommended. The EMA’s Committee for Medicinal Products for Human Use…