Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...
Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...
Charley Seckler has found "humor in the madness" of rolling through life with a rare disease. He drops a blog post weekly, ...
Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.
Dyne Therapeutics Inc. is advancing novel therapeutics for people living with genetically driven neuromuscular diseases.
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