Search for an orphan drug
Other search option(s)
28 Result(s)
Substance(s)
- (S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt
- 2,2-dimethylbutyric acid, sodium salt
- 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridin-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride
- 4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid
- Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
- Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene
- Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
- Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
- Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene
- Benserazide hydrochloride
- Bitopertin
- Deferasirox
- Deferiprone
- Hepcidin mimetic
- Hepcidin mimic peptide
- Human apotransferrin
- Luspatercept
- Sirolimus
- Synthetic hepcidin
- 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
- synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues