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Efficacy of Idebenone on Respiratory Function in Patients with Duchenne Muscular Dystrophy not using Glucocorticoids (DELOS): A Double-Blind Randomised Placebo-Controlled Phase 3 Trial
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The Ever-Expanding Spectrum of Congenital Muscular Dystrophies
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Spinal Muscular Atrophy A Timely Review
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Diagnosis and New Treatments in Muscular Dystrophies
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Diagnosis and Management of Motor Neurone Disease
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Monoclonal Antibody Therapies and Neurologic Disorders
Arch Neurol 65:1162-1165, Novack,J.C.,et al., 2008
Molecular Basis of the Neurodegenerative Disorders
NEJM 340:1970-1980, Martin,J.B., 1999
Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy
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The Muscular Dystrophies
BMJ 317:991-995, Emery,A.E.H., 1998
Investigation of Muscle Disease
JNNP 60:256-274, Mastaglia,F.L.&Laing,N.G., 1996
Trinucleotide Repeat Expansion in Neurological Disease
Ann Neurol 36:814-822, LaSpada,A.R.,et al, 1994
Molecular Genetics in Neurology
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