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

• প্রধান লেখক: Alexa C. Klimchak (লেখক), Lauren E. Sedita (লেখক), Louise R. Rodino-Klapac (লেখক), Jerry R. Mendell (লেখক), Craig M. McDonald (লেখক), Katherine L. Gooch (লেখক), Daniel C. Malone (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: Taylor & Francis Group, 2023-12-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: 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...