SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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Spinal muscular atrophy (SMA) refers to a group of autosomal recessive disorders characterized by degeneration of anterior horn cells in the spinal cord resulting in a progressive muscular weakness and atrophy. Our study is focused on the assessment of clinical course in patients with SMA types. To characterize the natural history of SMA 2 patients to gain further insight into the clinical course, we conducted a prospective observational cohort study of children and young adults with SMA 2. Patients were evaluated every 6 months over an 18 moth period.

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