Marisa Wexler, MS,  senior science writer—

Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).

Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD). The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European…

The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by muscular dystrophy (MD). One big underlying issue for the families of those with Duchenne and Becker muscular dystrophies is that the pandemic has caused many patients to…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…

Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to enroll 75 boys between the ages of 4 and 7 in the EMBARK trial in its latest announcement. Sarepta Therapeutics and Roche have announced the launch of EMBARK,…

Treatment with the investigational cell therapy CAP-1002 significantly improved arm and heart function in boys and young men at advanced stages of Duchenne muscular dystrophy (DMD), according to final data from the HOPE-2 clinical trial. The Phase 2 trial also “met various skeletal and cardiac endpoints [goals] suggesting clinically…

BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular dystrophy type 2i (LGMD2i), has been given fast track designation by the U.S. Food and Drug Administration (FDA). This designation aims to facilitate and speed the development and regulatory review of…

A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells — at targeting muscle cells may prove useful in developing a more effective gene therapy for muscular dystrophy (MD), according to researchers. In fact, this new vector “is more than…

Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy or OPMD, a type of the genetic disease that typically first affects the eyelids and the throat. The…

The U.S. Food and Drug Administration (FDA) has granted fast track status to EDG-5506, Edgewise Therapeutics’ investigational oral therapy for Becker muscular dystrophy (BMD). This designation is given to experimental treatments that are intended to treat serious conditions and fill an unmet medical need. It confers certain benefits, like…