Marisa Wexler, MS,  senior science writer—

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…

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…

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…

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…

Reduced heart function is associated with a higher rate of mortality among people with Duchenne muscular dystrophy (DMD), a new study reports. Results, however, did not suggest that reduced heart function is an independent predictor of survival for DMD patients. Instead, analyses indicated patients who took diuretics or who…

Treatment with glucocorticoids and heart medicines are key factors in determining the prognosis of Duchenne muscular dystrophy (DMD), according to a study. Results also highlighted genetics as an important factor affecting DMD progression. The paper, “Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review…

Despite broad initiatives seeking improvements, the time it takes an individual with Duchenne muscular dystrophy (DMD) to get a correct diagnosis has remained largely unchanged over the past three decades, a study reports. It still takes more than two years, on average, from the time symptoms are first noticed…

Treatment with the breast cancer medicine tamoxifen improved the functionality and survival of heart muscle cells in a cellular model of Duchenne muscular dystrophy (DMD), a study reports. The study, “Tamoxifen treatment ameliorates contractile dysfunction of Duchenne muscular dystrophy stem cell-derived cardiomyocytes on bioengineered substrates,” was published…

The Spectrum Health Helen DeVos Children’s Hospital (HDVCH) in Michigan has been recognized as providing the highest quality of care for patients with Duchenne muscular dystrophy. Located in Grand Rapids, the hospital has become the first in the state to receive a certification from Parent Project Muscular Dystrophy…