Pioneering Gene Therapy Market Entry for DMD: Sarepta has secured the first FDA approval for a gene therapy (ELEVIDYS) targeting Duchenne muscular dystrophy (DMD), establishing a significant first-mover advantage and a leading position in this high-need therapeutic area.

Established Exon-Skipping Franchise: With multiple FDA-approved exon-skipping therapies for DMD, Sarepta possesses a strong foundation of commercialized products and deep expertise in RNA-targeted treatments for neuromuscular diseases.

Robust Rare Disease Pipeline: Beyond its existing commercial products, Sarepta maintains a diverse and promising pipeline of gene therapies and RNA-based treatments for various forms of muscular dystrophies, indicating future growth potential.