Struggling With Muscle Weakness? It May Be A Sign Of Muscular Dystrophy
Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...
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Duchenne Muscular Dystrophy Space on Cusp of Pivotal Era
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
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Boys with DMD given Elevidys two years ago still showing motor gains
Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...
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Muscular Dystrophy
In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...
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Roche reports positive topline outcomes from DMD treatment trial
Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...
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Roche’s Elevidys demonstrates significant benefits for DMD patients in Phase 3
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
Sarepta Therapeutics' Duchenne Gene Therapy Shows Sustained Benefits And Disease Stabilization At Two Years
Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...
FDA Grants Orphan Drug Designation to IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy
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 ...
Science Daily29d
New study uncovers key insights into protein interactions in Duchenne muscular dystrophy, paving way for more targeted therapies
Biophysical characterization of the dystrophin C-terminal domain: Dystrophin interacts differentially with dystrobrevin isoforms. Journal of Biological Chemistry , 2024; 300 (12): 108002 DOI: 10. ...
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Duchenne Muscular Dystrophy
Although the drug increases dystrophin production, which would predict improvement in muscle function, this has not yet been shown. The first gene therapy to be approved for DMD, delandistrogene ...