Rare Diseases
Rare diseases are rare individually but in aggregate they are common; 1 in 17 people have a rare disease1.
Therapeutic areas within Rare Diseases
Get more familiar with the science of rare diseases. Learn about pathophysiology, testing and treatments, including specialised care services
Fabry Disease
See Everything 
Pompe Disease
See Everything 
MPS I Disease
See Everything 
Gaucher Disease
See Everything - Rare Diseases. National Center for Advancing Translational Sciences. Available at: https://www.raredisease.org.uk/media/1588/the-rare-reality-an-insight-into-the-patient-and-family-experience-of-rare-disease.pdf Accessed August 2022.
MAT-IQ-2300012-V1-Jan2023