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Spinal Muscular Atrophy Disease Mechanisms and Therapies: A Comprehensive Clinical Overview
"Spinal muscular atrophy is a severe genetic neuromuscular disorder characterized by the progressive degeneration of alpha motor neurons in the spinal cord, leading to profound muscle weakness and atrophy. Understanding its molecular mechanisms—primarily driven by mutations in the SMN1 gene and modulated by the SMN2 gene—has paved the way for breakthrough disease-modifying therapies that fundamentally alter the natural history of this condition. Introduction to Spinal Muscular Atrophy and Genetic Architecture Spinal muscular atrophy (SMA) is one of the most common autosomal recessive monogenic disorders, historically recognized as a leading ge..."
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