Pharmacological management of spinal muscular atrophy

Abstract

Spinal muscular atrophy is an autosomal recessive inherited disease, which associate with SMN gene mutation. It presents with a wide range of symptoms from weakness, and fatigue of muscles to non-ambulatory, and death. Currently, there is no complete cure therapy. However, FDA has approved Nusinersen as a standard treatment. Additionally, with direct-to-genetic treatments, pharmacological management for spinal muscular atrophy has shown a promising future.