News

Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant accelerated approval to the company’s experimental gene therapy SRP-9001 for people with Duchenne muscular dystrophy (DMD) who are able to walk. “We are delighted to confirm that based on the feedback we received…

Treatment with dystrophin expressing chimeric (DEC) cells was safe and led to long-term cellular and functional improvements in the heart, respiratory, and leg muscles of a mouse model of Duchenne muscular dystrophy, a study found. The findings support “future clinical application of DEC as a safer and potentially more…

A coalition of six non-profits will host a patient-focused meeting in September for six forms of limb-girdle muscular dystrophy (LGMD). The Sept. 23 online event is free, open to the public, and will focus on LGMD 2A (R1), 2C (R5), 2D (R3), 2E (R4), 2F (R6), and 2I (R9).

The first patient has been dosed in a pivotal U.S. Phase 3 study evaluating the investigational cell therapy CAP-1002 in males with advanced Duchenne muscular dystrophy (DMD). The trial, called HOPE-3 (NCT05126758), will evaluate the effect of CAP-1002 in upper limb function, among other assessments, in about…

A Phase 2 clinical trial testing Edgewise Therapeutics’ investigational oral therapy EDG-5506 is recruiting boys and men with Becker muscular dystrophy (BMD). Called CANYON (NCT05291091), the study plans to enroll up to 66 BMD patients, ages 12–50, across 14 clinical sites in the U.S., U.K., and the…

Note: This story was updated July 12, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there…

The first participant has been dosed in the Phase 3 REACH clinical trial testing the safety and effectiveness of Fulcrum Therapeutics‘ experimental oral therapy losmapimod in people with facioscapulohumeral muscular dystrophy (FSHD). If results from the trial are positive, they’re expected to support applications for lospmapimod’s approval…

Using the results of a New York State pilot testing program as evidence, Parent Project Muscular Dystrophy (PPMD) submitted its proposal seeking the addition of Duchenne muscular dystrophy (DMD) to the nationwide newborn screening panel. The proposal nominates DMD for inclusion in the Recommended Uniform Screening Panel or RUSP…

Boys with Duchenne muscular dystrophy (DMD) who cannot walk and have advanced scoliosis are at a higher risk of vertebral fractures due to significantly reduced bone mineral density (BMD), especially in the lumbar region of the spine, a study shows. Their low bone density may be associated with immobilization…

The investigational cell therapy CAP-1002 continued to improve arm function in boys and young men with advanced Duchenne muscular dystrophy (DMD), according to the HOPE-2 open-label extension study. These additional upper limb improvements came after HOPE-2 participants were off CAP-1002 for a mean of about one year before…