STAT+: Duchenne Muscular Dystrophy Patients Face Unequal Access to Promising Therapy

Exon-skipping drugs offer new hope for Duchenne muscular dystrophy patients, but access remains severely limited to only a fortunate few. These innovative therapies represent a critical test of the FDA's commitment to advancing rare disease treatments. The stark disparity in access raises important questions about equity and affordability in precision medicine. Patients and families are forced to navigate complex eligibility criteria and astronomical costs, highlighting the challenges in delivering breakthrough therapies to all who need them. This situation underscores the ongoing tension between innovation and accessibility in orphan drug development.