Breaking down barriers together
Through engagement with scientists, clinicians, industry, and regulators, Take Down Duchenne advocates to advance care for all individuals with dystrophin mutations
Toward universal genetic therapy
No genetic therapy approved in Duchenne muscular dystrophy is safe and effective for all.
Take Down Duchenne supports efforts to produce a universal full-length dystrophin replacement technology, as well as development of novel antisense oligonucleotide medicines.
Improving care for all
As the Duchenne disease process advances, therapeutic needs evolve.
Take Down Duchenne advocates for adolescents and adults with dystrophin mutations to have access to all available cardiopulmonary and other essential care.
Girls and women with dystrophin mutations are currently understudied and undertreated, yet their health may be affected.
