SMA THERAPIES II AND BIOMARKERS

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SMA is an autosomal recessive neuromuscular disorder characterized by degeneration of alpha motor neurons (AHCs) in the spinal cord and brain stem leading to muscular atrophy and weakness. Prognostic and predictive biomarkers are needed to inform SMA treatment. Neurofilaments (NFs) have been proposed as biomarkers in disorders characterized by axonal degeneration, although their clinical utility has not been established. NFs are intermediate filaments uniquely expressed in neuronal cells and are integral components of the cytoskeleton.

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