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"

8 matching diseasesClear search ×

Propionic acidemia

Ketotic hyperglycinemia · Propionic aciduria

ORPHA:35

Fumaric aciduria

Fumarase deficiency

ORPHA:24

Hydroxykynureninuria

Kynureninase deficiency · Xanthurenic aciduria

ORPHA:79155

Malonic aciduria

Malonyl-CoA decarboxylase deficiency · Malonic acidemia

ORPHA:943

Mevalonic aciduria

Complete mevalonate kinase deficiency · MVA

ORPHA:29

Organic aciduria

ORPHA:289899

Primary hyperoxaluria type 1

Glycolic aciduria · Peroxisomal alanine-glyoxylate aminotransferase deficiency

ORPHA:93598

Urocanic aciduria

Encephalopathy due to urocanase deficiency

ORPHA:210128