Some types of muscular dystrophy typically affect only males; some people with MD enjoy a normal life span with mild symptoms that progress very slowly; others experience swift and severe muscle ...
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...
Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...
A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...
The dysferlin protein is primarily responsible for repairing cell membranes. People with certain mutations in the gene coding for dysferlin develop muscular dystrophy—a group of muscle wasting ...
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
The dysferlin protein is primarily responsible for repairing cell membranes. People with certain mutations in the gene coding for dysferlin develop muscular dystrophy – a group of muscle wasting ...
IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new muscle with full length dystrophin in Becker Muscular Dystrophy(BMD). IPS ...
GlobalData on MSN13h
Roche reports positive topline outcomes from DMD treatment trialRoche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys for ...
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