skip to Main Content
Congratulations To Other Rare Disease Groups And Patients

Congratulations to other rare disease groups and patients

Today we learn that treatment for other rare diseases were approved for cystic fibrosis in Scotland and Batten disease. Thoughts and congratulations go out to everyone affected by or working hard to receive treatment for these diseases from TreatSMA.

Great news today that another two rare disease treatments received approval from the Scottish Medicines Consortium for cystic fibrosis and NICE for Batten disease, the latter being on an MAA basis like Spinraza. Also like Spinraza that these drugs had been rejected previously due to the high cost so these are further landmark decisions and evidence that the NHS is now beginning to focus on rare diseases. Both the Scottish Medicines Consortium and NHS England confirmed that they had negotiated confidential discounted agreements with the providers. You can read more information about both on the following links:

Batten disease

Cystic fibrosis 

Again, best wishes to everybody involved from TreatSMA

Back To Top