Entrada Therapeutics has selected ENTR-601-45 as an investigational therapy candidate for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 45 skipping. The company is planning to file an Investigational New Drug application in the second half of 2024. These applications are used to request…
The  U.S. Food and Drug Administration (FDA) is reviewing vamorolone for Duchenne muscular dystrophy (DMD) and has set a target action date of Oct. 26 for its decision on whether to approve the dissociative corticosteroid treatment. The regulatory agency accepted a new drug application (NDA) for the experimental…
Satellos Bioscience has designated SAT-3153 as a potential muscle regeneration treatment for Duchenne muscular dystrophy (DMD). The therapeutic candidate now will be tested in pre-investigational new drug (pre-IND) enabling studies intended to provide evidence to obtain regulatory approval for conducting a clinical trial. “We are thrilled to be in…
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…
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…
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