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
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Pompe Disease
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MPS I Disease
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Gaucher Disease
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References
- 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