Parents call on FDA for path forward after Duchenne’s gene therapy pause | Morning in America

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.