Orphanet: Search for an orphan drug
Search for an orphan drug
36 Result(s)
(-)-trans-3-(5,6-dihydro-4H-pyrrolo[3,2,1-ij]quinolin-1yl)-4-(1H-indol-3-yl) pyrrolidine-2, 5-dione
(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate
16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7-amino acid peptide
18-(p-(131 I)-iodophenyl)octadecyl phosphocholine
4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide
7-ethyl-10-hydroxy-camptothecin
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
Anlotinib
Autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4
Autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1
Brostallicin
Camsirubicin
Crenolanib besylate
Deforolimus
Doxorubicin hydrochloride (liposomal)
Doxorubicin(6-maleimidocaproyl)hydrazone
Fenretinide
Fibromun
Genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor
Human/murine chimeric monoclonal antibody against endoglin
Larotrectinib
Mammalian target of rapamcyin (mTOR) inhibitor
N-acetylsarcosyl-glycyl-L-valyl-D-allo-isoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-prolyl-N-ethylamide
Olaratumab
Ombrabulin
Paclitaxel
Palifosfamide
Propranolol hydrochloride
Sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein
Trabectedin
Vinorelbine tartrate
Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10
haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate