News

The U.S. Food and Drug Administration (FDA) has approved vamorolone for treating people with Duchenne muscular dystrophy (DMD), ages 2 and older. The therapy will be marketed as Agamree. “We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with…

A gene editing approach can restore dystrophin production in cell and animal models of Duchenne muscular dystrophy (DMD), according to its researchers. The study, “Targeting Duchenne Muscular Dystrophy by Skipping DMD Exon 45 with Base Editors,” was published in Molecular Therapy Nucleic Acids. DMD is caused by…

After three years of virtual academies, applications are now open for Duchenne Patient Academy 2023, an in-person training event for patient advocates for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The academy, presented by the World Duchenne Organization (WDO) and the Duchenne Data…

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…