Spinal muscular atrophy and Zolgensma

Reports have appeared in the UK media of the NHS administering the most expensive drug in the world to a 5-month-old boy. Onasemnogene abeparvovec (sold as Zolgensma), costing around two and a half million US-dollars (£1.79 million),* is a gene therapy agent to treat spinal muscular atrophy. The headlines quoted the price but gave scantContinue reading “Spinal muscular atrophy and Zolgensma”