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Retricted access

Research article

First published 2015-04

Detection of Survival Motor Neuron Protein in Buccal Cells Through Electrochemiluminescence-Based Assay

Hannes Steinkellner, Julia Etzler, Bernhard M. Gmeiner [...]

ASSAY and Drug Development Technologies

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Other

First published 2003-01

Development of a Gene Therapy Strategy for the Restoration of Survival Motor Neuron Protein Expression: Implications for Spinal Muscular Atrophy Therapy

Christine J. DiDonato, Robin J. Parks, Rashmi Kothary

Human Gene Therapy

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Research article

First published 2015-3

Association of Copy Numbers of Survival Motor Neuron Gene 2 and Neuronal Apoptosis Inhibitory Protein Gene With the Natural History in a Chinese Spinal Muscular Atrophy Cohort

Yu-jin Qu, Xiu-shan Ge, Jin-li Bai [...]

Journal of Child Neurology

Open access

Review article

First published 2020

Circulating microRNAs as potential biomarkers and therapeutic targets in spinal muscular atrophy

Tai-Heng Chen

Therapeutic Advances in Neurological Disorders

Open access

Research article

First published 2023-1-3

Disease Burden of Spinal Muscular Atrophy: A Comparative Cohort Study Using Insurance Claims Data in the USA

Julie Mouchet, Spyros Roumpanis, Eleni Gaki [...]

Journal of Neuromuscular Diseases

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Other

First published 2007-08

Animal Models of Spinal Muscular Atrophy

Aloicia Schmid, Christine J. DiDonato

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

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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Other

First published 2007-11

A Newborn With Spinal Muscular Atrophy Type 0 Presenting With a Clinicopathological Picture Suggestive of Myotubular Myopathy

Amelie Nadeau, Guy D'Anjou, Guillaume Debray [...]

Journal of Child Neurology

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

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