Muscular Dystrophy, Duchenne – Page 3

Duchenne Muscular Dystrophy: Potential Inclusion in Newborn Screening Programs

12 months ago talkbio0Tagged Decision Making, Diagnosis, Infant, Newborn, medical experts, Muscular Dystrophy, Duchenne

Duchenne muscular dystrophy, newborn screening, medical experts, decision-making, early diagnosis, treatment options.

Pfizer Temporarily Suspends Dosing in Advanced Duchenne Muscular Dystrophy (DMD) Trial After Patient Fatality

12 months ago talkbio0Tagged Clinical Trials, concerns, DMD, dosing, Duchenne, Evaluation, halt, late-stage study, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Pfizer, Safety, Suspensions, Transitory

Pfizer, Duchenne Muscular Dystrophy (DMD), clinical trial, dosing halt, patient death, late-stage study, temporary suspension, safety concerns, investigation.

Controversy Surrounds FDA’s Fast-Track Approval of Sarepta’s Duchenne Muscular Dystrophy Gene Therapy

12 months ago talkbio0Tagged Accelerated, Advocacy, Approved, Clinical Trials, DMD, Duchenne, Effectiveness, gene therapy, Muscular Dystrophy, Muscular Dystrophy, Duchenne, Regulation of biological process, Safety, United States Food and Drug Administration

FDA, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), gene therapy, accelerated approval, controversy, clinical trials, efficacy, safety, patient advocacy, regulatory process.