A few weeks back I got the opportunity to participate in the annual scientific meeting of the International Society for CNS Clinical Trials & Methodology. It was an interesting meeting to learn the key developments in the field and the Agency’s approval process. An interesting topic was around the pivotal clinical trial of Zolgensma, a gene therapy treatment for spinal muscular atrophy (SMA), for children under 2 years old with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma works by delivering a healthy copy of the SMN1 gene thereby, restoring the production of the SMN protein.