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...

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Bibliographic Details
Main Authors: Tamar E. Sztal, Emily A. McKaige, Caitlin Williams, Viola Oorschot, Georg Ramm, Robert J. Bryson-Richardson
Format: Article
Language:English
Published: BMC 2018-05-01
Series:Acta Neuropathologica Communications
Subjects:
Online Access:http://link.springer.com/article/10.1186/s40478-018-0546-9