DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in a Phase 1 trial, the treatment’s developer, PepGen, has announced. The single-ascending dose trial is investigating the treatment’s safety and tolerability in about 40 healthy adult males in Canada.
Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…
Thirteen new hospitals and health institutions have been added to the Muscular Dystrophy Association’s MDA Care Center Network, expanding the network’s reach across the United States. These additions, which comprise more than 150 care centers, include 10 new grant awards as well as three care affiliates that have been…
Electrical muscle stimulation that mimics resistance training exercises improves muscle structure, function, and survival rates in a Duchenne muscular dystrophy (DMD) zebrafish model, a new study reveals. “The longstanding consensus in the muscular dystrophy field is that minimizing resistance training preserves muscle strength and mass because it lowers the…
When it comes to making decisions about how care goals should change for adolescent and young adults with Duchenne muscular dystrophy (DMD) as the disease progresses, informal conversations among family members are more common than discussions with clinicians, according to a study based on interviews with DMD patients and…
An ion channel called Piezo1 regulates muscle stem cell (MuSC) states and the number of cells that respond quickly to muscle injury, promoting muscle repair, a study in mice shows. Notably, this ion channel was found to be poorly activated in a mouse model of Duchenne muscular dystrophy (DMD), and…
Four boys with Duchenne muscular dystrophy (DMD) show improved motor function and walking ability three years after being given the experimental gene therapy SRP-9001 in a Phase 1 clinical trial, new data reveal. The findings were presented in a poster, titled “Phase 1/2A Trial of Delandistrogene…
Boys with Duchenne muscular dystrophy (DMD) treated with the experimental gene therapy SGT-001 in the IGNITE DMD clinical trial continue to show improvements in motor and lung function after two years, according to new data presented at this year’s Muscular Dystrophy Association (MDA) Annual Meeting. “What we see…
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