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View article: Risdiplam distributes and increases <scp>SMN</scp> protein in both the central nervous system and peripheral organs
Risdiplam distributes and increases <span>SMN</span> protein in both the central nervous system and peripheral organs Open
Spinal muscular atrophy ( SMA ) is a rare, inherited neuromuscular disease caused by deletion and/or mutation of the Survival of Motor Neuron 1 ( SMN 1) gene. A second gene, SMN 2 , produces low levels of functional SMN protein that are in…