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Substance(s)
- (Z)-3-(3-(3,5-bis(trifluorométhyl)phényl-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylhydrazide
- Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene
- Adeno-associated viral vector serotype S3 encoding human alpha-galactosidase A cDNA
- Adeno-associated virus serotype C102 containing the human GLA gene
- Agalsidase alfa
- Agalsidase beta
- Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
- Alpha galactosidase A
- Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha
- Besilesomab
- Lucerastat
- Migalastat chlorhydrate
- Pegunigalsidase alfa
- adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
- 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