Testing of therapies in a novel nebulin nemaline myopathy model demonstrate a lack of efficacy
Abstract 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 myo...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2018-05-01
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Series: | Acta Neuropathologica Communications |
Subjects: | |
Online Access: | http://link.springer.com/article/10.1186/s40478-018-0546-9 |