CT parents push for newborn screening for Duchenne muscular dystrophy theridgefieldpress.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from theridgefieldpress.com Daily Mail and Mail on Sunday newspapers.
John Paul Hopkins, 32, from North Wales is hoping to get his son Rory, 6, who was diagnosed with Duchenne muscular dystrophy in 2021, onto gene therapy like Elevidys.
The prospect of “accelerated approval” for a single-dose treatment for Duchenne muscular dystrophy has drawn criticism from some scientists and health research experts who say the FDA is loosening its standards.