Evidence-based clinical review Gene Therapy for Spinal Muscular Atrophy in 2026: Lessons from the Frontlines Estimated reading time: 15 minutes Abstract Background Gene transfer has fundamentally changed the natural history of 5q spinal muscular atrophy
Tag: spinal muscular atrophy
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
