Recent Advance in Disease Modifying Therapies for Spinal Muscular Atrophy

Recent Advance in Disease Modifying Therapies for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease characterized by progressive weakness and atrophy of skeletal muscles. With homozygous survival motor neuron 1 (SMN1) gene mutation, all SMA patients have at least one copy of the SMN2 gene, which provides an opportunity fo...

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Bibliographic Details
Main Authors: Li-Kai Tsai, Chen-Hung Ting, Yo-Tsen Liu, Cheng-Tsung Hsiao, Wen-Chin Weng, Society for Neurological Rare Disorders-Taiwan
Format: Article
Language:English
Published: Wolters Kluwer Medknow Publications 2024-07-01
Series:Acta Neurologica Taiwanica
Subjects:
adeno-associated viral vector
antisense oligonucleotide
disease modifying therapy
gene therapy
motor neuron disease
spinal muscular atrophy
Online Access:https://journals.lww.com/10.4103/ANT.33-3_Review
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