![The sights of Avalon Air Show 2025: Day Three [PHOTOS]](https://breakingdefense.com/wp-content/uploads/sites/3/2025/03/f-35-avalon-final-day-scaled-e1743079275404.jpg?#)
STAT+: Wave to submit Duchenne drug for approval after Phase 2 success
Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new therapeutic option for patients.
Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new therapeutic option for patients with the rare disease.
Duchenne muscular dystrophy is a condition that primarily affects young boys, starving their bodies of dystrophin protein required for muscle function. The Food and Drug Administration has said that producing even a small amount of dystrophin — less than 5% — should improve a patient’s health.
In Wave’s Phase 2 study, 11 patients taking the company’s treatment began producing an average of 7.8% of normal dystrophin, the company announced Wednesday. Children taking the drug, WVE-N531, also showed a statistically significant improvement in the time it took to rise from the floor, which Leerink Partners Joseph Schwartz noted was greater than Sarepta Therapeutics showed with its Elevidys gene therapy, which the FDA approved in 2023.
