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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Dettagli Bibliografici
Pubblicato in:Ann Clin Transl Neurol
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley and Sons Inc. 2019
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Research Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6562036/
https://ncbi.nlm.nih.gov/pubmed/31211167
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.774
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