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Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy
Nemaline myopathies are heterogeneous congenital muscle disorders causing skeletal muscle weakness and, in some cases, death soon after birth. Mutations in nebulin, encoding a large sarcomeric protein required for thin filament function, are responsible for approximately 50% of nemaline myopathy cas...
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| Опубликовано в: : | Acta Neuropathol Commun |
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| Главные авторы: | , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
BioMed Central
2018
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5977763/ https://ncbi.nlm.nih.gov/pubmed/29848386 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40478-018-0546-9 |
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