SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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Spinal muscular atrophy (SMA) is caused by mutations in the survival motor neuron 1 gene. Clinically, SMA manifests in various ranges of severity including progressive muscle weakness and loss of motor function. SMA types are characterized based on clinical severity. Several therapeutic strategies are currently under clinical investigation and the first drug was approved in 2016. Given the variable clinical phenotype of SMA, it is essential to determine the best outcome measures and identify prognostic factors to inform clinical trial design.

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