Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for Priority Review to be given. Explore more details here.
The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...
In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...
Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...
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 ...
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...
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