Maria Susan Varughese

Treating Smn2B/− spinal muscular atrophy (SMA) mice with commercially approved metabolism-targeting interventions leads to improved disease phenotypes

Abstract Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular condition that is increasingly recognized as a multi-systemic disorder in which metabolic dysfunction plays a central role. Reframing SMA pathology from a neurocentric to a metabolic perspective reveal...