News

The U.S. Food and Drug Administration has granted rare pediatric disease status to NS Pharma‘s NS-089/NCNP-02, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), the company announced. The designation is intended to spur treatments for any rare disease (those affecting fewer than 200,000 people in…

The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Italfarmaco Group’s application requesting that oral givinostat be approved to treat Duchenne muscular dystrophy (DMD). Priority review, which shortens the consideration period to six months from 10, is given to therapies with…

After a year of treatment with the experimental oral therapy EDG-5506 in a Phase 1 study, most men with Becker muscular dystrophy (BMD) show stable or improved motor function — a marked contrast to the disease’s natural course, where motor function worsens over time. One-year findings in the…

The U.S. Food and Drug Administration (FDA) has cleared Benitec Biopharma to start a Phase 1b/2a clinical trial of the investigational gene therapy BB-301 for swallowing difficulties associated with oculopharyngeal muscular dystrophy (OPMD). Dosing is expected to start later this year, following the rollover of participants in…

Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review in the U.S. and Europe for Duchenne muscular dystrophy (DMD). Under the deal, Catalyst will give vamorolone’s developer Santhera Pharmaceuticals an upfront cash payment of $75 million,…

The U.S. Food and Drug Administration (FDA) granted accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) for children ages 4 to 5 with Duchenne muscular dystrophy (DMD) who can walk and have a confirmed mutation in the DMD gene. The treatment is now named Elevidys (delandistrogene moxeparvovec-rokl). “The approval…

Three advocacy organizations are teaming up to run a clinical trial to test if efgartigimod, an approved treatment for certain autoimmune diseases, might let more people with Duchenne muscular dystrophy (DMD) benefit from gene therapies. CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD)…

Stem cells derived from the amniotic membrane of pregnant women after childbirth “could provide therapeutic benefits” for people with Duchenne muscular dystrophy (DMD), according to researchers in Japan. These stem cells, known as human mesenchymal stromal cells, were able to delay DMD progression and preserve muscle function in a…

In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s part of the company’s strategy to gain global approval of SRP-9001 (delandistrogene moxeparvovec) as a gene therapy for Duchenne muscular dystrophy (DMD). These DMD patients will be non-ambulatory, or…

The LELANTOS-1 Phase 3 clinical trial, assessing pamrevlumab in combination with corticosteroids in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, did not reach its main goal. FibroGen, the therapy’s developer, announced the top-line results from the trial, whose main objective was to assess changes in upper…