Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

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"Maybe the children will have two dolls instead of 30 dolls, you know, and maybe the two dolls will cost a couple of bucks more than they would normally," he said.

Duchenne Muscular Dystrophy is the most common genetic killer of children worldwide and currently has no cure. You can donate to Chasing Connor’s Cure here. Around 100 boys are born with DMD in ...