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 ×

Inherited Creutzfeldt-Jakob disease

Inherited CJD

ORPHA:282166

Congenital brain dysgenesis due to glutamine synthetase deficiency

Inherited GS deficiency · Inherited glutamine synthetase deficiency

ORPHA:71278

Inherited acute myeloid leukemia

Acute myeloid leukemia · AML

ORPHA:319465

Inherited ichthyosis

Genetic ichthyosis

ORPHA:183435

Mitochondrial DNA-related progressive external ophthalmoplegia

Maternally-inherited CPEO · Maternally-inherited chronic progressive external ophthalmoplegia

ORPHA:663