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Research article

First published 2014-2

Clinical Features and Molecular Characterization of a Patient With Muscle-Eye-Brain Disease

Madalina Raducu, Rocío P. Cotarelo, Rogelio Simón [...]

Journal of Child Neurology

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Research article

First published 2013-03

Miao Yu, Yonglin He, Kejian Wang [...]

Human Gene Therapy

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Research article

First published 2021-6

Andreea Nissenkorn, Keren Yosovich, Zvi Leibovitz [...]

Journal of Child Neurology

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Research article

First published 2006-11

Jerry R. Mendell, Daniel R. Boué, Paul T. Martin

Pediatric and Developmental Pathology

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Research article

First published 2014-06

Charles H. Vannoy, Lei Xu, Elizabeth Keramaris [...]

Human Gene Therapy Methods (Part B)

Open access

Review article

First published 2015

Céline Bouchet-Séraphin, Sandrine Vuillaumier-Barrot, Nathalie Seta

Journal of Neuromuscular Diseases