Overview

• Elevidys gene therapy now approved for all ages with Duchenne muscular dystrophy, regardless of mobility status.
• FDA's Peter Marks overruled objections from three expert review teams and two directors to expand approval.
• Critics argue the decision undermines scientific rigor and could erode trust in the FDA.
• The therapy, costing $3.2 million per patient, missed its primary trial goals but showed some secondary benefits.
• Families of patients advocate for the treatment, citing urgent need despite mixed clinical results.