Spinal Muscle Atrophy: A Costly Battle against a Rare Genetic Disorder Worth 16 Crore

Spinal muscle atrophy is a disease of inheritance due to homozygous deletion of survival motor neuron gene 1 (SMN1) in the brain stem and spinal cord indicated as slowly advancing muscular dysfunction and atrophy. The 95% of cases reported of spinal muscle atrophy (SMA) depict deletion of the SMN1 gene. Targeted therapeutic strategies to combat SMA mainly focus on enhancing the level of full-length SMN protein. Currently, three drugs approved by FDA are Nusinersen, Zolgensma, and Risdiplam for treating SMA. With the advent These drugs have transformed the therapeutic approach of managing the fatal genetic disorder, treating number of patients worldwide along with the active participation of the organizations raising funds for the affected patients