Found: 25
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Overcoming Cellular Immunity to Prolong Adenoviral-Mediated Gene Expression in Sciatic Nerve.
- Published in:
- Annals of the New York Academy of Sciences, 1999, v. 883, n. 1, p. 397, doi. 10.1111/j.1749-6632.1999.tb08601.x
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- Publication type:
- Article
Development, reliability and validity of the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS).
- Published in:
- 2011
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- Publication type:
- Abstract
Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre study.
- Published in:
- 2020
- By:
- Publication type:
- journal article
Invited Review. The potential for gene therapy in duchenne muscular dystrophy and other genetic muscle diseases.
- Published in:
- Muscle & Nerve, 1993, v. 16, n. 11, p. 1141, doi. 10.1002/mus.880161102
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- Publication type:
- Article
Mutations in STAMBP, encoding a deubiquitinating enzyme, cause microcephaly-capillary malformation syndrome.
- Published in:
- Nature Genetics, 2013, v. 45, n. 5, p. 556, doi. 10.1038/ng.2602
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- Publication type:
- Article
420. Hydrodynamic Limb Vein Delivery of Naked IGF-1 Plasmid DNA Provides Therapeutic Benefit in SOD1(G93A) Murine ALS Model.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S161, doi. 10.1016/j.ymthe.2006.08.484
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- Publication type:
- Article
SMA CARNI-VAL TRIAL PART II: A Prospective, Single- Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy.
- Published in:
- PLoS ONE, 2011, v. 6, n. 7, p. 1, doi. 10.1371/journal.pone.0021296
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- Publication type:
- Article
SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy.
- Published in:
- PLoS ONE, 2010, v. 5, n. 8, p. 1, doi. 10.1371/journal.pone.0012140
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- Publication type:
- Article
Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy.
- Published in:
- PLoS ONE, 2009, v. 4, n. 5, p. 1, doi. 10.1371/journal.pone.0005268
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- Publication type:
- Article
Medical management of muscle weakness in Duchenne muscular dystrophy.
- Published in:
- PLoS ONE, 2020, v. 15, n. 10, p. 1, doi. 10.1371/journal.pone.0240687
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- Publication type:
- Article
Alpha-Synuclein Loss in Spinal Muscular Atrophy.
- Published in:
- Journal of Molecular Neuroscience, 2011, v. 43, n. 3, p. 275, doi. 10.1007/s12031-010-9422-1
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- Publication type:
- Article
Functional consequences and structural interpretation of mutations of human choline acetyltransferase.
- Published in:
- Human Mutation, 2011, v. 32, n. 11, p. 1259, doi. 10.1002/humu.21560
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- Publication type:
- Article
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
- Published in:
- Muscle & Nerve, 2021, v. 63, n. 5, p. 668, doi. 10.1002/mus.27187
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- Publication type:
- Article
Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.
- Published in:
- 2018
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- Publication type:
- journal article
SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
- Published in:
- 2014
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- Publication type:
- journal article
SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
- Published in:
- Muscle & Nerve, 2014, v. 49, n. 2, p. 187, doi. 10.1002/mus.23904
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- Publication type:
- Article
Compound muscle action potential and motor function in children with spinal muscular atrophy.
- Published in:
- Muscle & Nerve, 2010, v. 42, n. 5, p. 703, doi. 10.1002/mus.21838
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- Publication type:
- Article
Validation of the Charcot-Marie-Tooth disease pediatric scale as an outcome measure of disability.
- Published in:
- Annals of Neurology, 2012, v. 71, n. 5, p. 642, doi. 10.1002/ana.23572
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- Publication type:
- Article
Survival Motor Neuron Protein Regulates Apoptosis in an in vitro Model of Spinal Muscular Atrophy.
- Published in:
- Neurotoxicity Research, 2008, v. 13, n. 1, p. 39, doi. 10.1007/BF03033366
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- Publication type:
- Article
Survival Motor Neuron Protein Regulates Apoptosis in an in vitro Model of Spinal Muscular Atrophy.
- Published in:
- Neurotoxicity Research, 2008, v. 13, n. 1, p. 39, doi. 10.1007/BF03033366
- By:
- Publication type:
- Article
A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity.
- Published in:
- Human Molecular Genetics, 1994, v. 3, n. 4, p. 579
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- Publication type:
- Article
Long-term persistence of plasmid DNA and foreign gone expression in mouse muscle.
- Published in:
- Human Molecular Genetics, 1992, v. 1, n. 6, p. 363
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- Publication type:
- Article
Correction: Alternating Hemiplegia of Childhood: Retrospective Genetic Study and Genotype-Phenotype Correlations in 187 Subjects from the US AHCF Registry.
- Published in:
- 2015
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- Publication type:
- Correction Notice
Alternating Hemiplegia of Childhood: Retrospective Genetic Study and Genotype-Phenotype Correlations in 187 Subjects from the US AHCF Registry.
- Published in:
- PLoS ONE, 2015, v. 10, n. 5, p. 1, doi. 10.1371/journal.pone.0127045
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- Publication type:
- Article
Cerebral Infarct in Pediatric Neuro-Behçet's Disease.
- Published in:
- Journal of Child Neurology, 2008, v. 23, n. 11, p. 1331, doi. 10.1177/0883073808318199
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- Publication type:
- Article