Current and emerging targeted therapies for spinal muscular atrophy.

Abstract excerpt

INTRODUCTION: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder caused by insufficiency or total absence of the survival motor neuron protein due to a mutation in the SMN1 gene. The copy number of its paralog, SMN2, influences disease onset and phenotype severity. Current therapeutic approaches include viral and non-viral modalities affecting gene expression. Regulatory-approved drugs...

Topics

• Humans
• Quality of Life
• Muscular Atrophy, Spinal
• Oligonucleotides, Antisense
• Genetic Therapy
• Mutation