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Article

Gene therapy in spinal muscular atrophy.

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie - 1 Nov 2023

Audic Frédérique

Abstract excerpt

Infantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic...

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PMID: 38043977
DOI: 10.1016/S0929-693X(23)00222-1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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