Marisa Wexler, MS, senior science writer —

The U.S. Food and Drug Administration (FDA) has given Pfizer the go-ahead to open U.S. sites for the Phase 3 CIFFREO clinical trial, testing the experimental gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The FDA had placed a hold on Pfizer’s application to start the trial in…

A new natural history study that seeks to collect information about how Becker muscular dystrophy (BMD) progresses in the absence of treatment is now enrolling. The study is being led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University, its primary sponsor. Also collaborating…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

When it comes to making decisions about how care goals should change for adolescent and young adults with Duchenne muscular dystrophy (DMD) as the disease progresses, informal conversations among family members are more common than discussions with clinicians, according to a study based on interviews with DMD patients and…

Four boys with Duchenne muscular dystrophy (DMD) show improved motor function and walking ability three years after being given the experimental gene therapy SRP-9001 in a Phase 1 clinical trial, new data reveal. The findings were presented in a poster, titled “Phase 1/2A Trial of Delandistrogene…

Boys with Duchenne muscular dystrophy (DMD) treated with the experimental gene therapy SGT-001 in the IGNITE DMD clinical trial continue to show improvements in motor and lung function after two years, according to new data presented at this year’s Muscular Dystrophy Association (MDA) Annual Meeting. “What we see…

Treatment with PF-06939926, an experimental gene therapy for Duchenne muscular dystrophy (DMD), was generally well-tolerated among ambulatory patients — boys able to walk — in a Phase 1B clinical trial. Data also hint at the efficacy of the gene therapy, being developed by Pfizer, with researchers noting that…

The investigational gene therapy SRP-9003 was well tolerated overall and improved measures of physical functioning for up to three years among children with limb-girdle muscular dystrophy type 2E (LGMD2E) in a small clinical trial. “The observed durable treatment effect provides proof-of-concept, and supports our further clinical assessment of…

The corticosteroid Emflaza outperforms prednisone at slowing disease progression in people with Duchenne muscular dystrophy (DMD), according to new research presented at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting being held this week in Nashville, Tennessee. The research was funded by PTC Therapeutics, which markets Emflaza. Corticosteroids…

Treatment with BBP-418 improved walking ability among people with limb-girdle muscular dystrophy type 2i (LGMD2i) in a Phase 2 clinical trial, early data show. “To date, people with LGMD2i have no approved disease-modifying treatment options. Many of these patients see their quality of life deteriorate rapidly and lose their…