Myotubular myopathy and the neuromuscular junction: a novel therapeutic approach from mouse models

SUMMARY Myotubular myopathy (MTM) is a severe congenital muscle disease characterized by profound weakness, early respiratory failure and premature lethality. MTM is defined by muscle biopsy findings that include centralized nuclei and disorganization of perinuclear organelles. No treatments current...

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Main Authors: James J. Dowling, Romain Joubert, Sean E. Low, Ashley N. Durban, Nadia Messaddeq, Xingli Li, Ashley N. Dulin-Smith, Andrew D. Snyder, Morgan L. Marshall, Jordan T. Marshall, Alan H. Beggs, Anna Buj-Bello, Christopher R. Pierson
Format: Article
Language:English
Published: The Company of Biologists 2012-11-01
Series:Disease Models & Mechanisms
Online Access:http://dmm.biologists.org/content/5/6/852