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"

5 matching diseasesClear search ×

AICA-ribosiduria

5-amino-4-imidazole carboxamide ribosiduria · ATIC deficiency

ORPHA:250977

Formiminoglutamic aciduria

FTCD deficiency · Formiminotransferase cyclodeaminase deficiency

ORPHA:51208

GTP cyclohydrolase I deficiency

GTPCH deficiency · Hyperphenylalaninemia due to GTP cyclohydrolase deficiency

ORPHA:2102

Transaldolase deficiency

TALDO deficiency

ORPHA:101028

Transketolase deficiency

TKT deficiency · Short stature-developmental delay-congenital heart defect syndrome

ORPHA:488618