News

After 18 months, treatment with the experimental cell therapy CAP-1002 continues to show beneficial effects on upper limb function in people with Duchenne muscular dystrophy (DMD), according to new data from the open-label extension phase of the HOPE-2 study. The new trial data also suggests a consistent safety…

Regenxbio has started recruiting patients for the Phase 1/2 clinical trial to assess the safety and effectiveness of RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD). The trial, called AFFINITY DUCHENNE (NCT05693142), is expected to enroll 18 DMD patients ages 4 to 11 who…

The U.S. Food and Drug Administration (FDA) has given fast-track designation to AOC 1020, an investigational therapy for facioscapulohumeral muscular dystrophy (FSHD) developed by Avidity Biosciences. This designation allows more frequent interactions between a company and the regulatory agency, with the goal of speeding the development and review…

Pelvic floor disorders, including urinary and anal incontinence, are common in women with myotonic dystrophy type 1 (DM1) and a cause of significant distress, according to a recent study in Canada. “This study highlights the importance of screening these symptoms in a clinical setting and will help develop interventions…

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…