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FDA gives approval to first-ever gene therapy for Duchenne Muscular Dystrophy

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  • Staff Report 

The U.S. Food and Drug Administration has granted accelerated approval for the first-ever gene therapy, Elevidys, targeting Duchenne Muscular Dystrophy.


This groundbreaking development has been facilitated by research conducted in Rochester, and the therapy underwent phase three of its clinical trial at the University of Rochester Medical Center (URMC).

The approval will benefit pediatric patients aged four and five diagnosed with DMD, a debilitating condition that primarily affects boys, often leading to loss of mobility by their teenage years.


This gene therapy aims to replace the faulty gene responsible for the disease, marking a significant advancement from traditional symptomatic treatments.

Notably, participating families have reported significant improvements post-infusion.

Clinical trials continue as the makers of Elevidys work toward securing full approval for the therapy for children up to seven years old.



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