Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately 200,000 people in the US, often children. Now, researchers are studying a subtype of SMA, spinal muscular atrophy with respiratory distress type 1 (SMARD1), and have developed a gene replacement therapy that can be used to treat and control the disease in the future.

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