The Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA): Protocol for a Longitudinal Observational Study
BackgroundSpinal muscular atrophy (SMA) is an autosomal recessive genetic neuromuscular disorder with progressive muscle weakness and atrophy, mainly caused by lower motor neuron degeneration resulting from decreased levels of the survival motor neuron protein. Recently, 3 di...
Main Authors: | , , , , , , |
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Format: | Article |
Language: | English |
Published: |
JMIR Publications
2022-12-01
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Series: | JMIR Research Protocols |
Online Access: | https://www.researchprotocols.org/2022/12/e38878 |