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designated intractable/rare disease
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Stevens–Johnson syndrome
?
TBD
Skin disease
Rheumatoid arthritis
?
TBD
Type of autoimmune arthritis
Ulcerative colitis
?
TBD
Inflammatory bowel disease that causes ulcers in the colon
Parkinson's disease
?
TBD
Long-term neurodegenerative disease
Multiple sclerosis
?
TBD
Disease that damages the myelin sheaths around nerves
Autoimmune hemolytic anemia
?
TBD
Blood disorder in which antibodies target red blood cells
Tetralogy of Fallot
?
TBD
Type of congenital heart defect
Familial hypercholesterolemia
?
TBD
Genetic disorder characterized by high cholesterol levels
Tangier disease
?
TBD
Medical condition
Crohn's disease
?
TBD
Type of inflammatory bowel disease
ALS
?
TBD
Rare neurodegenerative disease
Cystic fibrosis
?
TBD
Genetic disorder inhibiting clearance of mucus from the lungs
Wilson's disease
?
TBD
Genetic multisystem copper-transport disease
