Understanding SMA: The Genetic Disease Changing with Gene-Based...

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2025-06-17 12:47:29

Understanding SMA: The Genetic Disease Changing with Gene-Based Therapies

Spinal Muscular Atrophy (SMA) is a severe, genetically inherited neuromuscular disorder characterized by progressive muscle atrophy and weakness due to degeneration of alpha motor neurons in the spinal cord. Once considered a leading genetic cause of infant mortality, SMA has undergone a radical transformation in recent years, driven by advances in genetic science and the development of...

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