Substance(s)

• (2'R,3'S)-2'-hydroxy-N-carboxy-3'-amino-5'-methyl-hexanoic,N-tert-butyl ester, 13 ester 5B-20-epoxy-1B,2a,4a,7B,9a,10a,13a-heptahydroxy-4,10-diacetate-2-benzoate-(1"S)-7,9-acrolein acetal-11(15-1)-abeotaxane
• 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4-c]pyridin-6-yl)ethan-1-one
• 17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5)
• 2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene
• 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid
• 3-Pentylbenzeneacetic acid sodium salt
• 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one
• 4,7,10,13,16,19-Docosahexaenoic acid
• 4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide
• 9-cis-Retinyl acetate
• Adeno-associated viral (AAV) vector expressing human retinoschisin-1 gene
• Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene
• Adeno-associated viral vector containing the human NADH dehydrogenase 4 gene (rAAV2/2-ND4)
• Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17-dimethylaminoethylamino-17-demethocygeldanamycin)
• Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
• Adeno-associated viral vector serotype 2 containing the human REP1 gene
• Adeno-associated viral vector serotype 2 containing the human Rab escort protein 1 gene
• Adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
• Adeno-associated viral vector serotype 5 containing the human ABCA4 gene
• Adeno-associated viral vector serotype 5 containing the human CHM gene
• Adeno-associated viral vector serotype 5 containing the human RLBP1 gene
• Adeno-associated viral vector serotype 8 containing the human AIPL1 gene
• Adeno-associated viral vector serotype 8 containing the human CNGA3 gene under the control of a cone arrestin promoter
• Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
• Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes
• Adeno-associated virus serotype 8 containing the human RdCVF sequence and the human RdCVFL sequence
• Adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene
• Adenovirus associated viral vector serotype 4 containing the human RPE65 gene
• Adenovirus associated viral vector serotype 5 containing the human RPE65 gene
• Adenovirus associated viral vector serotype 5 containing the human pde6 beta gene
• Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene
• Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (AAV2-hRPE65v2 )
• Adenovirus-associated viral vector serotype 8 containing the human RPGR gene
• Alglucerase
• All-cis-docosa-4,7,10,13,16,19-hexaenoic acid
• Allogeneic fetal human retinal progenitor cells expanded ex vivo
• Allogeneic human umbilical cord tissue-derived cells
• Allogeneic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth
• Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA
• Antisense oligonucleotide targeting exon 13 in the USH2A gene
• Antisense oligonucleotide targeting the USH2A gene
• Ataluren
• Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins
• Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins
• Davunetide
• Echothiophate iodide
• Ecothiopate iodide
• Emixustat hydrochloride
• Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotropic factor
• Expanded human allogeneic neural retinal progenitor cells extracted from neural retina
• Human Umbilical Tissue-Derived Cells
• Human embryonic stem-cell-derived retinal pigment epithelial cells
• Human plasminogen
• Humanised IgG4 monoclonal antibody against extracellular tau
• Imiglucerase
• Lentiviral vector containing the human ABCA4 gene
• Lentiviral vector containing the human MYO7A gene
• Ma09-Hrpe Cells
• Methotrexate
• Methylthioninium
• Mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene
• Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene
• Myriocin
• N-(3-(4-(3-(diisobutylamino)propyl)piperazin -1-yl)propyl)-1H-benzo[d]imidazol-2-amine disulphate
• Non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein
• Ramiprilat
• Recombinant adeno-associated viral vector containing the human CNGB3 gene
• Recombinant adeno-associated viral vector containing the human RPGR gene
• Recombinant adeno-associated viral vector containing the human retinoschisin gene
• Recombinant adeno-associated viral vector expressing the human CNGA3 gene
• Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene
• Recombinant human mesencephalic astrocyte-derived neurotrophic factor (MANF)
• Recombinant human methionine proinsulin
• Recombinant human nerve growth factor
• Recombinant human proinsulin
• Recombinant human rod-derived cone viability factor
• Recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326)
• Setmelanotide
• Soraprazan
• Tilavonemab
• Tolfenamic acid
• Unoprostone isopropyl
• adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene
• adenovirus associated viral vector serotype 5 containing the RPGR gene
• idebenone
• tideglusib

Tradename(s)

• CEREZYME
• CYSTARAN
• LUXTURNA
• RAXONE
• ZAVESCA