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Innovation in Rare Diseases
Rare Diseases
Patient Perspective
Timeline
Partners
What's Ahead
Patient Perspective
Find out what it's like to have a rare disease and how it has changed over time. You can read the personal story of a patient with Crohn's disease and learn what it was like to have leprosy in the Middle Ages.
Fabry Disease
Many Symptoms,
One Disease
Pulmonary Hypertension
Women with PAH Face Tough Decisions
Crohn's Disease
A Decade-Long Struggle with Crohn's: John's Story
Acromegaly
The "Gentle Giant" with
the Devastating Disease
Tuberculosis
A Disease from Antiquity
Leprosy
Reason for Exile in the
Old World
Progeria
Hope is in the Pipeline for Children with Progeria
ALS/Lou Gehrig's Disease
A Pitch for ALS
Cancer
Thankful for Another Day
Leukemia
The Wave of the Future Brings Hope