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"

4 matching diseasesClear search ×

Histidinemia

HAL deficiency · HIS deficiency

ORPHA:2157

3-hydroxy-3-methylglutaric aciduria

3-hydroxy-3-methylglutaryl-CoA lyase deficiency · HMG-CoA lyase deficiency

ORPHA:20

Adenylosuccinate lyase deficiency

ADSL deficiency · Adenylosuccinase deficiency

ORPHA:46

Cystathioninuria

Cystathionase deficiency · Gamma-cystathionase deficiency

ORPHA:212