Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy
Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy
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England: BioMed Central Ltd
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English
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England: BioMed Central Ltd
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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 cases. Despite the severity of the disease there is n...
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Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy
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TN_cdi_doaj_primary_oai_doaj_org_article_8cc6ba4097cf4c9bb514e650b9af4952
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_8cc6ba4097cf4c9bb514e650b9af4952
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2051-5960
E-ISSN
2051-5960
DOI
10.1186/s40478-018-0546-9