Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and one of the most common genetic causes of infant death. It is characterized by progressive weakness of the muscles, loss of ambulation, and death from respiratory complications. SMA is caused by the homozygous dele...

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Bibliographic Details
Main Authors: Tejal Aslesh, Toshifumi Yokota
Format: Article
Language:English
Published: MDPI AG 2022-01-01
Series:Cells
Subjects:
Online Access:https://www.mdpi.com/2073-4409/11/3/417