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Retricted access

Research article

First published 2004-3

Congenital Club Foot with Survival of Motor Neuron 1,Telomeric ( SMN1 ) Gene Deletion

Bernard Echenne, François Rivier, Agathe Roubertie [...]

Journal of Child Neurology

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Research article

First published 2013-11

Diabetic Ketoacidosis in an Adult Patient With Spinal Muscular Atrophy Type II

Nicole Holuba LaMarca, Lauren Golden, Rita Marie John [...]

Journal of Child Neurology

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Other

First published 1997-08

Gene Deletions in Arab Patients With Spinal Muscular Atrophy

Mohammad Z. Haider, Allie Moosa

Journal of Child Neurology

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Research article

First published 2012-4

The Natural History of Infant Spinal Muscular Atrophy in China

Xiushan Ge, Jinli Bai, Yanyu Lu [...]

Journal of Child Neurology

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Research article

First published 2013-05

Molecular Prenatal Diagnosis of Autosomal Recessive Spinal Muscular Atrophies Using Quantification Polymerase Chain Reaction

Wenting Jiang, Xing Ji, Yan Xu [...]

Genetic Testing and Molecular Biomarkers

Open access

Research article

First published 2025-11

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence

Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato [...]

Journal of International Medical Research

Open access

Review article

First published 2016

Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy

Saif Ahmad, Kanchan Bhatia, Annapoorna Kannan [...]

Neuroscience Insights

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Research article

First published 2018-11

Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells

Miaojin Zhou, Zhiqing Hu, Liyan Qiu [...]

Human Gene Therapy

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Research article

First published 2014-07

Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy

Marco A. Passini, Jie Bu, Amy M. Richards [...]

Human Gene Therapy

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Research article

First published 2002-06

Increased Survival and Function of SOD1 Mice After Glial Cell-Derived Neurotrophic Factor Gene Therapy

Gyula Acsadi, Roumen A. Anguelov, Huibin Yang [...]

Human Gene Therapy

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