학술논문
Recent therapeutic developments in spinal muscular atrophy.
Document Type
Article
Author
Source
Subject
*SPINAL muscular atrophy
*MOLECULAR pathology
*GENETIC disorders
*NEURODEGENERATION
*CLINICAL trials
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Language
ISSN
1300-0144
Abstract
Proximal spinal muscular atrophy (SMA) is an inherited neurodegenerative disease with a heterogeneous clinical phenotype. Although there is no cure for SMA, several strategies are currently being developed. In this review, we summarize the ongoing clinical trials and molecular mechanisms of successful approaches to SMA treatment. [ABSTRACT FROM AUTHOR]