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

• Main Authors: Alexa C. Klimchak (Author), Lauren E. Sedita (Author), Louise R. Rodino-Klapac (Author), Jerry R. Mendell (Author), Craig M. McDonald (Author), Katherine L. Gooch (Author), Daniel C. Malone (Author)
• Format: Book
• Published: Taylor & Francis Group, 2023-12-01T00:00:00Z.
• Subjects: article
• Online Access: 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...