Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...
Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...
(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...
Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...
Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...
Duchenne muscular dystrophy (DMD) is the most common type ... The muscle problems can cause cramps at times, but in general, DMD isn’t painful. Your child will still have control of their ...
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