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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…

Pliant Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) for permission to begin clinical testing of PLN-101325, its experimental therapy for muscular dystrophy, by the end of this year. In preclinical studies involving cell and animal models — the results of which were presented…

The experimental oral therapy EDG-5506 has been generally well-tolerated and lowered markers of muscle damage among men with Becker muscular dystrophy (BMD) in the open-label ARCH clinical trial, early data show. Findings supported the shift to a higher daily treatment dose in study patients. “These interim 2-month ARCH data…

Defeat Duchenne Canada is investing $1.14 million in three research projects aimed at providing better treatments for Duchenne muscular dystrophy, the most common type of muscular dystrophy and one that affects about 1 in 3,500 male births. The trio of three-year projects joins six other research efforts…

The Muscular Dystrophy Association (MDA) is hosting a one-day games streaming fundraiser, dubbed “MDA Rivals,” this Saturday, June 18. The event, streaming on MDA’s Twitch channel, will feature stars from MDA Let’s Play — a platform that aims to raise funds for MDA and awareness about muscular…

FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD). The LELANTOS-2 study (NCT04632940) has enrolled 73 boys with DMD, ages 6 to 12, who maintain their ability to walk.