Duchenne exon-skipping drugs: FDA rare-disease access test

Exon-skipping therapies for Duchenne muscular dystrophy remain accessible only to a subset of patients, highlighting a gap between FDA's rare-disease incentives and real-world availability. The article frames this as a critical test of FDA policy, but specifics on which drugs or access barriers are not detailed. This matters as it could influence regulatory and reimbursement debates for rare-disease treatments.