Search for an orphan drug
13 Result(s)
- (2S,3R,4R,5S)-1-[5-(2-fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol
- Acetylleucine
- Adeno-associated viral vector serotype 9 containing the human HEXA and HEXB genes
- Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
- Besilesomab
- Biphenyl-substituted L-ido configured deoxynojirimycin derivative
- N-Acetyl-L-Leucine
- Recombinant adeno-associated viral vector serotype 2/1 encoding human beta-hexosaminidase alpha and beta subunits
- Sinbaglustat
- Venglustat
- 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
