Table 2 Orphan diseases presented on the television series “House, M.D.” and corresponding orphan drug designations and approvals by the FDA, if present, as well as respective season and episodes
Orphan disease | ORPHA Number | Number of compounds with an orphan drug designation (by the FDA) | Number of compounds with an orphan drug approval (by the FDA) | FDA approved compounds | Season | Episode |
|---|---|---|---|---|---|---|
Sickle cell trait | 232 | 40 | 3 | Hydroxyurea, L-glutamine | 7 | 2 |
Mastocytosis | 98,292 | 8 | 3 | Cromolyn sodium, imatinib mesylate, midostaurin | 8 | 1 |
Muckle-Wells syndrome | 575 | 3 | 3 | Anakinra, Canakinumab, Rilonacept | 7 | 14 |
Ornithine Transcarbamylase Deficiency | 664 | 4 | 2 | Benzoate and phenylacetate, sodium phenylbutyrate | 1 | 15 |
Familial Mediterranean fever | 342 | 3 | 2 | Canakinumab, colchicine | 5 | 6 |
Fabry disease | 324 | 9 | 1 | Ceramide trihexosidase/alpha-galactosidase A | 6 | 3 |
African trypanosomiasis | 3385 | 4 | 1 | Eflornithine HCl | 1 | 7 |
Acute intermittent porphyria | 79,276 | 4 | 1 | Hemin | 1 | 22 |
Leprosy | 548 | 3 | 1 | Clofazimine | 1 | 13 |
Diffuse lepromatous leprosy | 548 | 3 | 1 | Clofazimine | 5 | 1 |
Wegener’s disease | 900 | 3 | 1 | Rituximab | 7 | 23 |
Chronic granulomatous disease | 379 | 2 | 1 | Interferon gamma 1-b | 3 | 8 |
Mucormycosis | 73,263 | 2 | 1 | Isavuconazonium sulfate | 8 | 14 |
Hereditary coproporphyria | 79,273 | 2 | 1 | Hemin | 5 | 10 |
Hereditary Angioedema | 91,378 | 2 | 1 | C1-esterase-inhibitor | 3 | 5 |
Thrombotic thrombocytopenic purpura | 54,057 | 7 | 0 | – | 1 | 19 |
Hereditary hemorrhagic telangiectasia | 774 | 3 | 0 | – | 3 | 16 |
Senile amyloidosis | 330,001 | 3 | 0 | – | 3 | 3 |
Alport syndrome | 63 | 2 | 0 | – | 8 | 7 |
Giant cell arteritis | 397 | 1 | 0 | – | 8 | 12 |
Von Hippel-Lindau syndrome | 892 | 1 | 0 | – | 4 | 2 |
Wiskott-Aldrich Syndrome | 906 | 1 | 0 | – | 5 | 15 |
Subacute sclerosing panencephalitis | 2806 | 1 | 0 | – | 1 | 2 |
Variegate porphyria | 79,473 | 1 | 0 | – | 7 | 10 |
Ehlers-Danlos Syndrome | 98,249 | 1 | 0 | – | 7 | 18 |
Langerhans cell histiocytosis | 389 | 0 | 0 | – | 3 | 10 |
MERRF syndrome | 551 | 0 | 0 | – | 3 | 7 |
Henoch-Schönlein Purpura | 761 | 0 | 0 | – | 6 | 18 |
Adult Refsum disease | 773 | 0 | 0 | – | 7 | 17 |
Kawasaki’s syndrome | 2331 | 0 | 0 | – | 8 | 5 |
Reye’s syndrome | 3096 | 0 | 0 | – | 8 | 9 |
Takayasu’s arteritis | 3287 | 0 | 0 | – | 3 | 15 |
Whipple’s disease | 3452 | 0 | 0 | – | 6 | 15 |
Bartonella | 50,839 | 0 | 0 | – | 7 | 16 |
McLeod syndrome | 59,306 | 0 | 0 | – | 7 | 12 |
Rickettsialpox | 83,312 | 0 | 0 | – | 7 | 7 |
Miller Fisher syndrome | 98,919 | 0 | 0 | – | 8 | 16 |
Polyglandular autoimmune syndrome type III | 227,982 | 0 | 0 | – | 8 | 13 |
Hughes-Stovin syndrome | 228,116 | 0 | 0 | – | 6 | 11 |
Marburg multiple sclerosis | 228,157 | 0 | 0 | – | 7 | 8 |
Arnold-Chiari malformation | 268,882 | 0 | 0 | – | 6 | 19 |
Primary antiphospholipid syndrome | 398,097 | 0 | 0 | – | 6 | 5 |