The Muscular Dystrophy Association is hosting its 2026 Clinical & Scientific Conference (March 8–11 in Orlando), where several companies developing therapies for Duchenne muscular dystrophy (DMD) are presenting their research findings.
Defeat Duchenne Canada is pleased to highlight important updates for the Canadian Duchenne community.
Avidity Biosciences
Avidity Biosciences shared their results from their global phase 3 study of del-zota for individuals amenable to exon 44 skipping. Data showed near-normal creatine kinase (CK) levels, improvements in functional outcomes, and reductions in inflammation and fibrosis in muscle.
Capricor Therapeutics
Capricor Therapeutics shared results from their phase 3 HOPE-3 trial of Deramiocel, a cell therapy designed to help preserve heart and skeletal muscle function. The therapy works by reducing inflammation and scarring in muscle tissue, which may help protect muscle health in people living with Duchenne.
Dyne Therapeutics
Dyne Therapeutics shared results from their phase 1/2 DELIVER trial of DYNE-251, an exon-51 skipping therapy. Participants showed improvements in lung function and heart performance compared to the expected decline seen in Duchenne, with a generally favorable safety profile.
Precision Biosciences
Precision Biosciences shared their new preclinical data on PBGENE-DMD, a gene-editing therapy. In laboratory models, the treatment restored production of a near full-length dystrophin protein across multiple muscle groups, including heart muscle, and improved muscle function.
REGENXBIO
REGENXBIO shared new data on their investigational gene therapy RGX-202 and hosted a symposium focused on improving the design and interpretation of Duchenne gene therapy clinical trials.
Santhera Pharmaceuticals
Santhera Pharmaceuticals shared results from the Guardian study showing that AGAMREE had similar effectiveness to traditional corticosteroids (prednisone or deflazacort) in delaying loss of walking ability, with fewer side effects such as fractures, cataracts, and growth suppression.
Sarepta Therapeutics
Sarepta Therapeutics shared long-term follow-up data from their phase 3 EMBARK study of their gene therapy delandistrogene moxeparvovec, including offering complementary perspectives on treatment outcomes by a caregiver and safety data from studies with up to 7.5 years of follow-up.
Satellos Biosciences
Satellos Biosciences shared their phase 1 study results of SAT-3247 in healthy volunteers and adults with Duchenne. This oral drug is designed to help regenerate skeletal muscle that is lost due to the disease. Results suggest the drug is safe and effective in all study participants.
Solid Biosciences
Solid Biosciences shared a clinical update on their SGT-003 gene therapy program. Results revealed robust expression of microdystrophin and restoration of proteins that work with dystrophin to improve muscle health. This is also confirmed by lower levels of several proteins measured in the blood including CK, ALT, AST, LDH, and titin suggesting a reduction in muscle cell injury.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: