We are so happy to announce that NHSE and Novartis Gene have reached an agreement which will enable families in England to have access to gene therapy for Spinal Muscular Atrophy - one of the most expensive treatments in England on the NHS.
This was our first sitting on a panel when approving a drug along with two other SMA Charities, stakeholders and professionals.
Like Spinraza there will be stipulations as to who can receive it. Please read further by clicking here