Rare Disease Library

Browse Orphanet-curated rare diseases with HPO phenotype annotations, synonyms, and linked support programs. Search by name, abbreviation, or synonym.

Try: "IgA nephropathy", "C3G", "Berger's disease", "PKU", "Duchenne"

7 matching diseasesClear search ×

Fabry disease

Alpha-galactosidase A deficiency · Anderson-Fabry disease

ORPHA:324

Addison disease

Primary Addison disease · Autoimmune adrenalitis

ORPHA:85138

Alexander disease

AxD

ORPHA:58

Chylomicron retention disease

Anderson disease · CMRD

ORPHA:71

Farber disease

Acid ceramidase deficiency · Farber lipogranulomatosis

ORPHA:333

Glycogen storage disease due to glycogen branching enzyme deficiency

GSD due to glycogen branching enzyme deficiency · GSD type 4

ORPHA:367

Wilson disease

Hepatolenticular degeneration

ORPHA:905