Ans: I will tell about another genetic disease called “Spinraza” which is considered as the one of the rarest disease which affects the motor neurons in infants which causes death in infants. It is a genetic condition and is known as spinal muscular atrophy which has a severe and chronic condition with no cure. Actually the drugs for treating the disease are called Spinraza which have great effect on this disease. As the disease has no cure method, gene therapy was the only option for treating this disease.