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Spinal Muscular Atrophy

Prevalence: ~60K

neuromuscular

Official·OMIM:253300 Official·ORPHA70

Genetic disease leading to motor neuron loss. Caused by mutations in the SMN1 gene.

Approved Therapies

Zolgensma (onasemnogene abeparvovec)

FDA Approved5/24/2019AAV Gene Therapy

Associated Genes

SMN1Survival Motor Neuron 1

Chromosome 5Autosomal Recessive