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Vemurafenib improves muscle histopathology in a mouse model of LAMA2-related congenital muscular dystrophy

Laminin-α2-related congenital muscular dystrophy (LAMA2-CMD) is a neuromuscular disease affecting around 1-9 in 1,000,000 children. LAMA2-CMD is caused by mutations in the LAMA2 gene resulting in the loss of laminin-211/221 heterotrimers in skeletal muscle. LAMA2-CMD patients exhibit severe hypotoni...

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Detalles Bibliográficos
Autores principales: Oliveira-Santos, Ariany, Dagda, Marisela, Wittmann, Jennifer, Smalley, Robert, Burkin, Dean J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10184677/
https://www.ncbi.nlm.nih.gov/pubmed/37021539
http://dx.doi.org/10.1242/dmm.049916