Recent therapeutic developments in spinal muscular atrophy

Objective & Hypothesis: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.

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Key Findings & Data: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.

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Conclusion & Impact:In this review, we summarize the ongoing clinicaltrials and molecular mechanisms of successful approaches to SMA treatment.

🔬Design:Review & Systematic Survey

📍Scope:Comprehensive Sample

⚙️Variables:Domain Parameters

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 clinicaltrials and molecular mechanisms of successful approaches to SMA treatment.

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Cite This Article

BORA, G., TOPALOĞLU, H. A., KURT, C. E. B., KAYMAZ, A. Y., HALİLOĞLU, V. G., YURTER, H. E., ÖZDAMAR, S. E. (2022). Recent therapeutic developments in spinal muscular atrophy. Turkish Journal of Medical Sciences, Cilt: 48 - Sayı: 2, 203–211. https://ajindex.com/articles/610390

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