News
Our work focuses on bringing to light the needs of people with rare and complex diseases and ensuring that they are able to engage with all forms of research that might enhance their lives
NICE approves life-changing medication for treating MPS IVA Morquio
We are delighted to announce that Elosulfase alfa (Vimizim®) has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA
A Consensus Statement: Psychological Support at Diagnosis
Approximately 6% of the population is affected by a rare disease. The diagnostic odyssey is significant, and patients and their families can often be left
Optimising Success for Access to Rare Disease Treatments
If you missed our latest webinar on the changing landscape of Highly Specialised Technologies in the UK it’s not too late to catch up. View
