Spinal muscular atrophy (SMA) is a rare genetic disorder that affects the motor neurons in the spinal cord, resulting in muscle weakness and gradual loss of movement. It is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to a deficiency in the survival motor neuron protein (SMN) which is critical for the normal functioning and survival of motor neurons. SMA is the cause of more infant deaths worldwide than any other genetic disorder.
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