Abstract: Journal of Clinical Case Studies and Family Medicine

Abstract:
This content introduces spinal muscular atrophy (SMA) gene therapy with onasemnogene abeparvovec, focusing on its development, mechanism of action, and impact on patients. Loss of motor neuronee is a genetic condition known as SMA, and onasemnogene abeparvovec aims to provide a working copy of the gene for survival motor neuronee SMN. The article explores the severity grades of SMA, the current treatment landscape, management strategies, and nutritional challenges faced by patients. It explores the economic considerations of gene therapy, comparing costs with other SMA treatments. Safety profiles, adverse events, patient selection criteria, and clinical studies are also discussed. The document concludes with insights into the future direction of SMA therapy, emphasising ongoing advancements and emerging treatments like Zolgensma.