Sarepta Therapeutics: Revolutionizing the Treatment of Rare and Infectious Diseases

Sarepta Therapeutics is a biotechnology company that focuses on developing innovative RNA-based therapies to treat rare and infectious diseases. The company's mission is to revolutionize the treatment of these diseases by leveraging the power of RNA to create new possibilities for patients.
Sarepta's product portfolio includes:
1. Exondys 51: A therapy for Duchenne muscular dystrophy, a rare genetic disorder that affects muscle strength and function.
2. Vyondys 53: A therapy for Fabry disease, a lysosomal storage disorder that can lead to kidney damage, heart problems, and other complications.
3. Golodirsen: A therapy for myotonic dystrophy, a genetic disorder that causes muscle weakness and wasting.
4. SRP-9001: An investigational therapy for the treatment of Pompe disease, a rare genetic disorder that affects muscle strength and function.
Sarepta's technology platform is based on RNA-targeting therapies, which work by silencing specific disease-causing genes. The company's approach has the potential to treat a wide range of rare and infectious diseases, and its pipeline includes several other product candidates in various stages of development.
Overall, Sarepta Therapeutics is a biotech company that is dedicated to developing innovative RNA-based therapies to improve the lives of patients with rare and infectious diseases.