A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and help regenerate muscle tissue in a mouse model of the disease. The researchers are now seeking…
News
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD). “The submission of the BLA marks a pivotal step for Capricor and those impacted by…
During 2024, Muscular Dystrohy News Today covered the latest scientific studies, advances in treatment, and clinical trials for the multiple types of muscular dystrophy (MD). Here is a list of the top 10 most-read stories we published last year. No. 10 – Patient death prompted pause in DMD…
The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases. Altogether, the funding totals more than $5 million across 21 grants, according to a press release…
The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD) treated in a Phase 2 trial, according to top-line results announced by the therapy’s developer, Edgewise Therapeutics. “This landmark study presents compelling biomarker data and promising signals that…
Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) patients, 4 and older. The recommendation came from the U.K.’s National Institute for Health and Care Excellence (NICE) and follows Agamree’s approval…
The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who are working to develop new treatments for facioscapulohumeral muscular dystrophy (FSHD). “This competition will bring together the brightest minds in medicine, technology, and science, all working toward a cure for this devastating disease,”…
Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in preclinical studies and eased symptoms in a mouse model of myotonic dystrophy type 1 (DM1). The company presented the findings at the Cell Symposia: Chemical Biology in Drugging the Undrugged conference, held earlier…
Sarepta Therapeutics is acquiring from Arrowhead Pharmaceuticals the exclusive global rights to develop ARO-DUX4 and ARO-DM1, two RNA interference (RNAi) therapeutic candidates in Phase 1/2 clinical testing, each for one type of muscular dystrophy. ARO-DUX4 is being tested for facioscapulohumeral muscular dystrophy (FSHD) and ARO-DM1 for…
A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got pregnant, which led to a healthy outcome for her and her baby girl, according to researchers in Japan. The woman’s case was reported in “Successful Pregnancy Outcome With Preconception…
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