Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints

OBJECTIVE: Limb girdle muscular dystrophy type R9 (LGMD R9) is an autosomal recessive muscle disease for which there is currently no causative treatment. The development of putative therapies requires sensitive outcome measures for clinical trials in this slowly progressing condition. This study ext...

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Detalles Bibliográficos
Autores principales: Murphy, Alexander P., Morrow, Jasper, Dahlqvist, Julia R., Stojkovic, Tanya, Willis, Tracey A., Sinclair, Christopher D. J., Wastling, Stephen, Yousry, Tarek, Hanna, Michael S., James, Meredith K., Mayhew, Anna, Eagle, Michelle, Lee, Laurence E., Hogrel, Jean‐Yves, Carlier, Pierre G., Thornton, John S., Vissing, John, Hollingsworth, Kieren G., Straub, Volker
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6562036/
https://www.ncbi.nlm.nih.gov/pubmed/31211167
http://dx.doi.org/10.1002/acn3.774

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