Impaired renal function in patients with spinal muscular atrophy: A longitudinal cohort study
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BackgroundSpinal muscular atrophy (SMA) is caused by loss-of-function of the survival motor neuron 1 (SMN1) gene and deficiency of the ubiquitously expressed SMN protein. Genetic therapies can partially rescue motor units and improve prognosis of SMA, but effe... ...
