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 ×

Mild phenylketonuria

Mild PKU · mPKU

ORPHA:79253

Classic phenylketonuria

Classic PKU · PKU

ORPHA:79254

Infantile Refsum disease

IRD · Mild peroxisome biogenesis disorder-Zellweger spectrum disorder

ORPHA:772

Maternal phenylketonuria syndrome

Hyperphenylalaninemic embryopathy · Maternal hyperphenylalaninemia

ORPHA:2209

Mild hyperphenylalaninemia

Mild HPA · Non-PKU HPA

ORPHA:79651

Phenylketonuria

PKU

ORPHA:716

Tetrahydrobiopterin-responsive phenylketonuria

BH4-responsive phenylketonuria · BH4-responsive PKU

ORPHA:293284