Financial Performance - Preliminary total net product revenue of 1.79 billion for full-year 2024, exceeding full-year guidance by over 384.2 million for Q4 2024 and 60 million[4] - RNA-based PMO net product revenue totaled 967.2 million for full-year 2024[4] - Year-end 2024 cash, cash equivalents, restricted cash, and investments balance was approximately 2.9 to $3.1 billion[4] Product Information - ELEVIDYS is a gene transfer therapy for Duchenne muscular dystrophy, addressing the underlying genetic cause through micro-dystrophin production[5][6] - The company has over 40 programs in various stages of development, focusing on precision genetic medicine for rare diseases[18] Regulatory and Safety Considerations - Continued approval for ELEVIDYS in non-ambulatory patients may depend on verification of clinical benefit in confirmatory trials[7] - The company emphasizes the importance of monitoring for adverse reactions following ELEVIDYS administration, including liver injury and myocarditis[11][15]
Sarepta Therapeutics(SRPT) - 2024 Q4 - Annual Results