Sarepta Therapeutics develops and commercializes gene therapy, antisense oligonucleotide, and RNA interference therapies targeting rare neuromuscular and genetic diseases with limited existing treatment options. The company's commercial products include ELEVIDYS, a gene therapy for Duchenne muscular dystrophy, and three exon-skipping therapies (EXONDYS 51, VYONDYS 53, and AMONDYS 45) approved for specific DMD patient populations, along with several product candidates in development for conditions such as limb-girdle muscular dystrophy, myotonic dystrophy, and facioscapulohumeral muscular dystrophy. Sarepta generates revenue through direct product sales in the United States via specialty pharmacies, expanded access programs in select countries, and strategic partnerships with other pharmaceutical companies, while benefiting from orphan drug exclusivity and patent protections in its ultra-rare disease markets.