Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review by Tanya von Reuss, Mark Gubar, Alex Avisoni and Nik Resovski April 8, 2021September 8, 2022 Novartis revealed the most expensive drug in the world. And it is amazing.