Sarepta's DMD gene therapy secures FDA approval at $3.2M cost.

Sarepta Therapeutics has received preliminary approval from the FDA for its gene therapy for Duchenne muscular dystrophy, a rare muscle-wasting disease that almost always affects males. The treatment will cost $3.2m for a one-time treatment, and will be mostly paid by insurers. The FDA has approved the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition.