Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

• Glavni autori: Alexa C. Klimchak (Autor), Lauren E. Sedita (Autor), Louise R. Rodino-Klapac (Autor), Jerry R. Mendell (Autor), Craig M. McDonald (Autor), Katherine L. Gooch (Autor), Daniel C. Malone (Autor)
• Format: Knjiga
• Izdano: Taylor & Francis Group, 2023-12-01T00:00:00Z.
• Teme: article
• Online pristup: Connect to this object online.

ABSTRACTBackground: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectiveness analyses are important to help contextualize the...