Qatar Tribune on MSN1h
Sidra establishes Gene Therapy Center for rare genetic diseases
Tribune News NetworkDohaSidra Medicine, a member of Qatar Foundation, has established a Gene Therapy Center to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular ...
Muscular Dystrophy News7h
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 ...
The American Journal of Managed Care7h
EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
manilatimes9h
MDA Launches 75th Anniversary Campaign and Premieres New PSA to Shine a Light on Rare Diseases
MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...
PMLiVE11h
Roche/Sarepta announce two-year results for Duchenne muscular dystrophy gene therapy
Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...
biopharma-reporter13h
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 ...