Explainer: Decoding spinal muscular atrophy
SMN protein is produced inadequately leading to severely compromised bodily functions, or physical disability. There have been reports in the Indian news media recently about the plight of infants afflicted by Spinal Muscular Atrophy (SMA), a genetic neuromuscular disorder. One of the recognised remedies – Zolgensma (Onasemnogene Abeparvoves-Xioi), an FDA-approved gene therapy medicine, costing INR […]