Insights into spinal muscular atrophy from molecular biomarkers.
Neural Regen Res
; 2024 Jun 26.
Article
in En
| MEDLINE
| ID: mdl-38934395
ABSTRACT
ABSTRACT Spinal muscular atrophy is a devastating motor neuron disease characterized by severe cases of fatal muscle weakness. It is one of the most common genetic causes of mortality among infants aged less than 2 years. Biomarker research is currently receiving more attention, and new candidate biomarkers are constantly being discovered. This review initially discusses the evaluation methods commonly used in clinical practice while briefly outlining their respective pros and cons. We also describe recent advancements in research and the clinical significance of molecular biomarkers for spinal muscular atrophy, which are classified as either specific or non-specific biomarkers. This review provides new insights into the pathogenesis of spinal muscular atrophy, the mechanism of biomarkers in response to drug-modified therapies, the selection of biomarker candidates, and would promote the development of future research. Furthermore, the successful utilization of biomarkers may facilitate the implementation of gene-targeting treatments for patients with spinal muscular atrophy.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Language:
En
Journal:
Neural Regen Res
Year:
2024
Document type:
Article
Affiliation country:
China