The approved antiparasitic medicine pentamidine, loaded into biocompatible and biodegradable nanoparticles, successfully eased signs of myotonic dystrophy type 1 (DM1) in cell-based models, a study shows. The researchers noted that these findings show the potential of nanoparticles to deliver therapeutic agents to treat muscle cells in people with DM1…
News
The U.S. Food and Drug Administration (FDA) has placed a hold on clinical testing of ENTR-601-44, Entrada Therapeutics‘ investigational therapy for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 44 skipping. The decision was in response to the company’s investigational new drug (IND) application,…
Wave Life Sciences‘ experimental exon-skipping therapy WVE-N531 appears to be working as expected, according to early data from a small clinical trial of the therapy in a first group of three boys with Duchenne muscular dystrophy (DMD). “These data provide early evidence that WVE-N531 is … leading to…
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…
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,…
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