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Parents call on FDA for path forward after Duchenne's gene therapy pause

Parents call on FDA for path forward after Duchenne's gene therapy pause

Yahoo28-07-2025
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient receiving treatment for muscular dystrophy. Alison and William Small are urging the agency to restore access to the drug, the only FDA-approved treatment for Duchenne's muscular dystrophy. Speaking to 'Morning in America,' the Smalls call for transparency and a clear path forward from federal regulators. They say families affected by Duchenne's deserve to understand the risks and that many believe the treatment is worth fighting for.
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