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 ×

Thrombotic microangiopathy

TMA

ORPHA:93573

Severe primary trimethylaminuria

TMAU

ORPHA:468726

Gerstmann syndrome

ORPHA:221117

Gerstmann-Straussler-Scheinker syndrome

Subacute spongiform encephalopathy, Gerstmann-Straussler type

ORPHA:356

Guttmacher syndrome

Preaxial deficiency-postaxial polydactyly-hypospadias syndrome

ORPHA:2957

Kostmann syndrome

Infantile agranulocytosis · Severe congenital neutropenia type 3

ORPHA:99749

Torpedo Maculopathy

Solitary hypopigmented nevus of the retinal pigment epithelium · TM

ORPHA:674935