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AI Summary & Key Takeaways
AI Insight⚡ TL;DR(Too Long; Didn't Read)
In this review, we summarize the ongoing clinical trials and molecular mechanisms of successful approaches to SMA treatment.
📌 3 Key Takeaways
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Objective & Hypothesis:Proximal spinal muscular atrophy (SMA) is an inherited neurodegenerative disease with a heterogeneous clinical phenotype.
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Key Findings & Data:Although there is no cure for SMA, several strategies are currently being developed.
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Conclusion & Impact:In this review, we summarize the ongoing clinical trials and molecular mechanisms of successful approaches to SMA treatment.
🔬Design:Review & Systematic Survey
📍Scope:Comprehensive Sample
⚙️Variables:Spinal muscular atrophy · therapeutic approaches · clinical trials
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.
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Cite This Article
BORA, G., YEŞBEK-KAYMAZ, A., BEKİRCAN-KURT, C. E., HALİLOĞLU, V. G., TOPALOĞLU, H. A., ERDEM-YURTER, H., ERDEM-ÖZDAMAR, S. (2018). Recent therapeutic developments in spinal muscular atrophy. Turkish Journal of Medical Sciences, Cilt: 48 - Sayı: 2, 203–211. https://ajindex.com/articles/112963