News

Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. Three boys with Duchenne muscular dystrophy (DMD) treated with the experimental cell therapy DT-DEC01 in a clinical trial continue to show improvements in motor…

Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in a proof-of-concept clinical trial continue to show improvements in motor function four years after dosing. That’s according to new data discussed this month at the Muscular Dystrophy Association’s MDA Clinical…

People with Duchenne muscular dystrophy (DMD) who are treated with Exondys 51 (eteplirsen) live significantly longer than would be expected without the treatment, by a median of more than five years, according to a new analysis. The longest survival times were generally seen among patients who started on…

Researchers are planning to enroll a new group of patients in a natural history study to collect data about how Duchenne muscular dystrophy (DMD) progresses and affects a person’s life. Natural history studies don’t test an experimental therapy, but instead work to collect detailed information on how a disease…

In a bid to further understand and solve potential gene therapy safety concerns in Duchenne muscular dystrophy (DMD), the Muscular Dystrophy Association (MDA) has awarded two research grants totaling nearly $400,000. The funding will allow the awarded researchers and their teams to develop new strategies to prevent immune system…

A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular dystrophy (LGMD), a new study reports. “We show an effective and precise repair to correct the most frequent [LGMD type 2A] mutation … Our approach is a promising source for…

Protecting structures on the end of chromosomes, called telomeres, by increasing levels of the protein TRF2, was found to improve heart cell survival and function in a model of Duchenne muscular dystrophy (DMD), a new study reports. “Our findings suggest that preserving telomere lengths plays a critical role in…

Sarepta Therapeutics is calling for applications for the 6th annual Route 79, The Duchenne Scholarship Program, for the 2023-2024 academic year. The scholarships, of up to $5,000, will be awarded to up to 20 students living with Duchenne muscular dystrophy (DMD), and up to five siblings of…

The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9), being developed by Asklepios BioPharmaceutical (AskBio). This designation is given to therapies for life-threatening or chronically debilitating diseases — those affecting no more than five…

GIVI-MPC, a stem cell therapy to create new muscle in Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status encourages the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S.