8 March 2021 - A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy is set to become the most expensive treatment ever approved by NICE.
NICE’s draft guidance published today recommends £1.79 million treatment Zolgensma (onasemnogene abeparvovec, Novartis Gene Therapies) for babies aged up to 12 months with type 1 spinal muscular atrophy..
This is one of the severest forms of the inherited condition and the life-expectancy of people with it is usually less than 2 years.