Prospective cohort study of spinal muscular atrophy types 2 and 3 in Spanish population


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 2 and 3. To characterize the natural history of SMA 2 and 3 to gain further insight into the clinical course. We conducted a prospective observational cohort study of children and young adults with SMA 2 and 3.


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