Treatment with AOC 1001 eased myotonia, when muscles are unable to relax after a contraction, in adults with myotonic dystrophy type 1 (DM1), according to early data from the Phase 1/2 MARINA clinical trial. MARINA data also showed — in all treated participants — AOC 1001 was successfully delivered…
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PGN-EDODM1, a therapy candidate for myotonic dystrophy type 1 (DM1), showed a favorable safety profile in preclinical studies, including those in non-human primates, PepGen, the company developing the treatment, announced. This work is expected to support a request to initiate a single ascending dose clinical trial of PGN-EDODM1…
The first patients have consented to share their clinical data with the Electronic Health Record (EHR) Study, according to an announcement from Parent Project Muscular Dystrophy (PPMD). The study’s goal is to help clinicians and researchers improve care and learn more about Duchenne and Becker muscular dystrophy. The…
Sarepta Therapeutics‘ investigational GALGT2 gene therapy helped stabilize muscle function when delivered at a higher dose to a boy with Duchenne muscular dystrophy (DMD), data from a Phase 1/2a clinical trial show. The other trial participant, a boy with more severe disease, had a slight lung function improvement…
CITGO raised $2.3 million this year to help advance research, improve patient care, and bolster advocacy efforts for the neuromuscular community, continuing its long-standing support for the Muscular Dystrophy Association (MDA), . Throughout the year, employees of the Houston, Texas-based petroleum company, along with vendors, contractors, marketers,…
The U.S. Food and Drug Administration (FDA) has given priority review Sarepta Therapeutics‘ application seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec), the company’s experimental gene therapy for Duchenne muscular dystrophy (DMD) in patients who can walk. An FDA decision is expected on or before May 29, 2023. “We are delighted…
Blocking a calcium channel called TRPC6 prolonged survival in a mouse model of Duchenne muscular dystrophy (DMD) by improving skeletal and heart muscle function and reducing bone deformities, a study showed. Its results support clinical trials of TRPC6 inhibitors in Duchenne patients as “a novel DMD therapy,” the researchers wrote,…
Analyzing the type of mutation in the DMD gene combined with assessing motor milestones may help better distinguish Duchenne muscular dystrophy (DMD) from Becker muscular dystrophy (BMD) early in life, a study found. Combining both approaches had better predictive power than using either alone, and could mean that children will…
PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed promising effects in preclinical studies, the therapy’s developer PepGen announced. The company also announced promising results from cell experiments for two other investigational exon-skipping therapies, PGN-EDO45 and PGN-EDO44.
Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001 (delandistrogene moxeparvovec) — its experimental gene therapy for Duchenne muscular dystrophy (DMD) — in patients with pre-existing antibodies against the gene therapy’s viral carrier. Preclinical work conducted by…
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