Results of a Two-Year Pilot Study of Clinical Outcome Measures in Collagen VI-related Myopathy and LAMA2-related Muscular Dystrophy

Potential therapies are currently under development for two congenital muscular dystrophy (CMD) subtypes: Collagen VI Related Myopathy (COL6-RM) and Laminin α2-related dystrophy (LAMA2-RD). However, appropriate clinical outcome measures to be used in clinical trials have not been validated in CMDs....

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Detalhes bibliográficos
Publicado no:Neuromuscul Disord
Main Authors: Meilleur, Katherine G., Jain, Minal S., Hynan, Linda S., Shieh, Ching-Yi, Kim, Eunice, Waite, Melissa, McGuire, Michelle, Fiorini, Courtney, Glanzman, Allan M., Main, Marion, Rose, Kristy, Duong, Tina, Bendixen, Roxanna, Linton, Melody M., Arveson, Irene C., Nichols, Carmel, Yang, Kelly, Fischbeck, Kenneth H., Wagner, Kathryn R., North, Kathryn, Mankodi, Ami, Grunseich, Christopher, Hartnett, Elizabeth J., Smith, Michaele, Donkervoort, Sandra, Schindler, Alice, Kokkinis, Angela, Leach, Meganne, Foley, A. Reghan, Collins, James, Muntoni, Francesco, Rutkowski, Anne, Bönnemann, Carsten G
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5125840/
https://ncbi.nlm.nih.gov/pubmed/25307854
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2014.09.010
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