Joana Vindeirinho, PhD,  —

Five of seven clinical milestones in managing Duchenne muscular dystrophy (DMD) occurred significantly later for Black males than for white males, a new study reports. These gaps appeared to stem from the initial delays in the first evaluation, which occurred for Black children 2.3 years later than for white…

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…

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…