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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 (Â鶹Éç). 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 (Â鶹Éç) who can walk and have a confirmed mutation in the Â鶹Éç 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 (Â鶹Éç) benefit from gene therapies. CureDuchenne, Muscular Dystrophy Association (Â鶹Éç), 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 (Â鶹Éç), according to researchers in Japan. These stem cells, known as human mesenchymal stromal cells, were able to delay Â鶹Éç 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 (Â鶹Éç). These Â鶹Éç 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 (Â鶹Éç) 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…

MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (Â鶹Éç), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). A Phase 2 trial testing MP1032 in Â鶹Éç patients is expected to start next year. “Currently, Â鶹Éç cannot be cured, but it can be treated,”…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1 study of PGN-EDODM1, an experimental medicine by PepGen for people with myotonic dystrophy type 1 (DM1). A clinical hold is an order issued by the FDA to delay…

Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for myotonic dystrophy type 1 (DM1). The designation is meant to support the development of potential therapies for rare, life-threatening, or chronically debilitating diseases. The benefits include reduced fees, clinical…

The U.S. Food and Drug Administration (FDA) has pushed back its decision on SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (Â鶹Éç). A decision is now expected by June 22, according to a press release. If the agency decides to approve SRP-9001, it’s expected to be…