Orphanet: Search for an orphan drug
Search for an orphan drug
66 Result(s)
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid
(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one
(R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate
(S)-1-(4-(1-(3,4,5-trimethoxyphenyl)-1H-imidazol-4-ylamino)thieno[2,3-d]pyrimidin-2-yl)pyrrolidine-2-carboxamide
2-[4-[3-(methylamino)-1-phenylpropoxy]phenyl]ethanol hydrochloride
4-(3-cyano-6-ethoxyquinolin-2-yl)-N- (2-fluorophenyl)-1,4-diazepane-1-carbothiomide
4-(6-(4-(piperazin-1-yl)phenyl_pyrazolo[1,5-a]pyrimidin-3-yl)quinoline hydrochloride
5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride
Acamprosate calcium
Acebutolol
Afatinib
Allogeneic cultured postnatal thymus-derived tissue
Alpha-tocopherol and ascorbic acid
Bacterial lipase
Balipodect
Beloranib
Blarcamesine
Cannabidiol
Cannabidiol acid methyl ester
Cannabidivarin
Carbetocin
Codergocrine mesilate, oxitriptan
Davunetide
Diazoxide choline
Gefitinib
Human monoclonal antibody against activin A
IPN-60130
Ibudilast
Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene
Liprotamase
Lisuride Maleate
Lyophilized microbiota purified from the stool of screened, healthy human donors
Maralixibat chloride
Mavoglurant
Melatonin
Metadoxine
Metreleptin
N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine
Oxytocin
Palovarotene
Psilocybine
R-baclofen
Recombinant human insulin-like growth factor-I/recombinant human insulin-like growth factor binding protein-3
Recombinant microbial lipase
Replication-incompetent adeno-associated virus serotype 9 vector encoding the human fragile x mental retardation 1 gene
Setmelanotide
Somatropin
Sulindac
Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin
Trofinetide
[5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride
cyclo-L-glycyl-L-2-allylproline
tideglusib