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SMA Drug Development Gains Momentum Under NINDS Model for Neuro‐Research Partnerships

aegis of the NINDS. SMA results from mutations in a gene called SMN1, resulting in abnormally low levels of survival motor neuron (SMN) protein. Without a normal amount of SMN, motor neurons in the spinal cord degenerate, causing muscle atrophy, loss of strength, disability, and premature death due to respiratory complications. An estimated 7.5 million Americans, about 1 in 40 people, carry the gene mutation that causes SMA, but