Advances in the Therapy of Spinal Muscular Atrophy

Abstract

Spinal muscular atrophy (SMA) has been the prototypical neurological disorder for designing and validating disease-specific therapies. There are now 3 approved treatments for SMA, each using a novel therapeutic modality, and each effective in changing the natural history of SMA, a condition that was previously universally fatal in childhood in its severe infantile form. By changing the natural history of this disease, new phenotypes and new complications, as well as new clinical, research, and ethical questions, are emerging.