nusinersen therapy for pediatric spinal muscular atrophy

nusinersen therapy for pediatric spinal muscular atrophy Study Background  Spinal muscular atrophy (SMA) is an inherited motor neuron disease caused by the degeneration of anterior horn cells of the spinal cord and brainstem. SMA primarily affects babies and young children and has an incidence of 1:10,000. It is caused by a homozygous

nusinersen therapy for pediatric spinal muscular atrophy