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Autor:
Amy D. Harper, Haluk Topaloglu, Eugenio Mercuri, Vasiliy Suslov, Liwen Wu, Cigdem Y. Ayanoglu, Michael Tansey, Michelle L. Previtera, Robert A. Crozier, Leslie Magnus, Paula R. Clemens
Publikováno v:
Scientific Reports, Vol 14, Iss 1, Pp 1-9 (2024)
Abstract Duchenne muscular dystrophy (DMD) is an X-linked recessive disease characterized by mutations in the dystrophin gene, causing motor and pulmonary function decline. Viltolarsen is indicated for patients with dystrophin gene mutations amenable
Externí odkaz:
https://doaj.org/article/9968d3b7d6514a56bdbd3bf8f3a6e367