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(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-uno (R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate (S)-1-(4-(1-(3,4,5-trimethoxyphenyl)-1H-imidazol-4-ylamino)thieno[2,3-d]pyrimidin-2-yl)pyrrolidine-2-carboxamide (S)-6-idrossi-2,5,7,8-tetrametil-N-((R)-piperidina-3-il)croman-2-carbossamide cloridrato 17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5) 17alpha,21-diidrossi-16alpha-metil-pregna-1,4,9(11)-triene-3,20-dione 18-mer antisense oligonucleotide complementary to SCN1A mRNA, sodium salt 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA 2'-O-metil-fosforotioato oligonucleotide 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine 2-[4-[3-(methylamino)-1-phenylpropoxy]phenyl]ethanol hydrochloride 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione 20- hydroxyecdysone 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine 3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone 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-(3-cyano-6-ethoxyquinolin-2-yl)-N- (2-fluorophenyl)-1,4-diazepane-1-carbothiomide 4-(6-(4-(piperazin-1-yl)phenyl_pyrazolo[1,5-a]pyrimidin-3-yl)quinoline hydrochloride 4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide 4-benzil-2-naftalen-1-il-1,2,4-tiadiazolidin-3,5-dione 5'-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-sp-dA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3' 5'-moemC-(sp)-moemC-(p)-moeA-(p)-moemC-(p)-moeG-(p)-moeA-(p)-dmC-(sp)-dA-(sp)-dT-(sp)-dA-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dmC-(sp)-moeT-(p)-moeA-(sp)-moemC-(sp)-moeA 3' 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate 9-cis-retinil acetato A DMD exon 44 skipping PMO and a proprietary cyclic peptide, EEV, that is designed to enable robust muscle tissue uptake, high cellular internalization and endosomal escape of oligonucleotide therapeutics Acebutolol Acetylleucine Acido 3,5-diiodotiropropionico Acido carglumico Adeno-associated viral vector LK03 encoding human methylmalonyl-CoA mutase Adeno-associated viral vector containing a modified U7-snRNA gene 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 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA Adeno-associated viral vector serotype 2 containing the human SLC6A3 gene Adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene Adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene Adeno-associated viral vector serotype 8 containing the human MD1 gene Adeno-associated viral vector serotype 8 containing the human MTM1 gene Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase Adeno-associated viral vector serotype 9 containing the human MECP2 gene Adeno-associated viral vector serotype 9 containing the human SLC13A5 gene Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase Adeno-associated viral vector serotype rh.10 expressing beta-galactosidase Adeno-associated virus serotype 2/8 vector containing the human PDE6A gene Adeno-associated virus serotype 5 containing the human NR2E3 gene Adeno-associated virus serotype 9 encoding human NGLY1 gene Adeno-associated virus serotype 9 expressing the cDNA for human MECP2 Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase Adeno-associated virus serotype PTC3 expressing the human UBE3A gene Adeno-associated virus serotype R100 containing the human RPGRorf15 gene isoform Adeno-associated virus serotype hu68 containing the human GLB1 gene Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene Adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene Adeno-associated virus vector serotype 9 encoding human gigaxonin gene Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter 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 8 containing the human RPGR gene Afatinib Alfa-N-acetilglucosaminidasi umana ricombinante All-cis-docosa-4,7,10,13,16,19-hexaenoic acid Allogeneic cultured postnatal thymus-derived tissue 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 Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase Alofuginone idrobromuro Alpha-L-iduronidase fused to Fab fragment of a humanised monoclonal antibody targeting human transferrin receptor Alpha-tocopherol and ascorbic acid Anticorpo Monoclonale ricombinante per il recettore dell'insulina umana fuso con iduronato 2 solfatasi Anticorpo monoclonale umanizzato anti-miostatina 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 Asp-Arg-Val-Tyr-Ile-His-Pro Ataluren Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene Autologous CD34+ enriched cells transduced with a self-inactivating lentiviral vector containing the codon-optimized RPS19 gene Autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human iduronate 2-sulfatase gene Bacterial lipase Balipodect Beloranib Benzoate/phenylacetate Benzoic acid, sodium salt Benzyl ethyl aminoazobenzene quaternary ammonium Beta-glucuronidasi umana ricombinante Betaine anhydrous Betamethasone Blarcamesine Brivaracetam Calcio acamprosate Cannabidiol acid methyl ester Cannabidiolo Cannabidivarin Carbetocina Cellule CD34+ autologhe trasdotte con un vettore lentivirale contenente il gene umano SGSH Cellule epatiche umane eterologhe (per infusione) Cellule progenitrici allogeniche della retina neurale espanse, estratte dalla retina neurale Cellule progenitrici eterologhe di fegato umano adulto Cellule staminali eterologhe di fegato umano adulto Ceramidasi acida ricombinante umana Chemically modified human recombinant sulfamidase Chenodeoxycholic acid Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 Clemizole hydrochloride Cloridrato dell'acido (1S,3S)-3-amino-4-(difluorometilene) ciclopentanecarbossilico Codergocrine mesilate, oxitriptan Codon -optimised human ornithine transcarbamylase mRNA complexed with lipid -based nanoparticle Copper histidinate Copper nanocluster conjugated to acetate, histidinate and ascorbate DNA plasmid encoding human transferrin gene Davunetide Desipramina cloridrato Dexamethasone sodium phosphate encapsulated in human erythrocytes Diazoxide choline Diidrato del sale di sodio della Genisteina Dimethyl fumarate Ecopipam Elamipretide HCl Emtricitabine Eparan N-solfatasi umana ricombinante Eteplirsen Everolimus Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor Ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast Exon 55 specific phosphorothioate oligonucleotide Fattore di crescita del tessuto nervoso ricombinante umano Felbamate Fenfluramina cloridrato Fenilbutirrato di sodio Fingolimod Fosdenopterin Gaboxadol monohydrate Ganaxolone Gefitinib Givinostat Gliceril-tri-(4-fenilbutirrato) Glycine, L-alanine, L-arginine, L-aspartic acid, L -cysteine, L-cystine, L- glutamic acid, L-histidine, L-lysine monohydrate, L -methionine, L- phenylalanine, L-proline, L-serine, L-threonine, L-tryptophan, L-tyrosine, taurine Golodirsen Govorestat Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) Human Umbilical Tissue-Derived Cells Human induced pluripotent stem cell derived myogenic progenitor cells (GIVI-MPC) Human monoclonal antibody against activin A Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase IPN-60130 Ibudilast Idebenone Idursulfase Idursulfase beta Insulin-like Growth Factor-1 Isopropile di unoprostone Ketamine Lamotrigine Laronidasa Linee cellulare encapsulate di pigmento retinale umano transfettate con un vettore plasmidico esprimente il fattore neurotrofico ciliare umano Liposomal mannose-1-phosphate Lisuride Maleate Live biotherapeutic consisting of metP and metDC genes which are incorporated into the genome of a probiotic strain of E. coli Nissie 1917 Lorcaserin Lyophilized microbiota purified from the stool of screened, healthy human donors Mavoglurant Mecasermin Mecasermin rinfabate Melatonina Mercaptamine-pantetheine disulfide Metformin and L-citrulline Methyl 4-((3S,5R,7R,8R,9S,10S,13R,14S,17S)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-3- ylmethyl)amino)butyl)amino)hexadecahydro-1H-cyclopenta[a]phenanthren-17-y1)pentanoate maleate Methyl(R)-4-((3S,5R,7R,8R,9S,10S,13R,14S,17R)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-3-ylmethyl)amino)butyl)amino)hexadecahydro-1H-cyclopenta[a]phenanthren-17-yl)pentanoate Metreleptin Modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671 Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles Modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles Modified messenger ribonucleic acid encoding human ornithine transcarbamylase enzyme encapsulated into lipid nanoparticles Modified version of a leucine decarboxylase enzyme from Planctomycetaceae bacterium Myriocin N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide N-Acetyl-L-Leucine N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine N-sulfoglucosamine sulfohydrolase fused to a humanised monoclonal antibody targeting the human transferrin receptor Naproxcinod Non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein Oligonucleotide fosforotioato specifico per l'esone 44 Oligonucleotide fosforotioato specifico per l'esone 45 Oligonucleotide fosforotioato specifico per l'esone 51 Oligonucleotide fosforotioato specifico per l'esone 53 Oligonucleotide fosforotioato specifico per l'esone 55 Olipudase alfa Omigapil maleate Ossitocina Palovarotene Pamrevlumab Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase) Pentosan polysulfate sodium Piridossal 5'-fosfato Poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with cystathionine gamma-lyase (Pyridoxal 5'-phosphate cofactor) Prednisone Proinsulina metionina umana ricombinante Proinsulina umana ricombinante Proteina di fusione recombinante consistente in un anticorpo monoclonale contro il recettorer dell' l'insulina umana fuso con alpha-L-iduronidasi Proteina ricombinante di fusione costituita dalla porzione extracellulare del recettore dell'activina umana di tipo IIB legata al dominio FC di IgG1 umana Psilocybine Pyridoxine and L-pyroglutamic acid R, S-O-(3-piperidino-2-idrossi-1-propil) - acido nicotinico dicloridrato amidoxime R-baclofene RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl] N,N-dimethylaminophosphonamidate RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[------], octahydrochloride Rasagiline Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase Recombinant adeno-associated viral vector containing the human RPGR gene Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter 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 adeno-associated viral vector serotype 9 carrying the gene for the human E6-AP ubiquitin protein ligase Recombinant adeno-associated viral vector serotype 9 containing human iduronidase gene Recombinant adeno-associated viral vector serotype 9 containing the human N-alpha-acetylglucosaminidase gene Recombinant adeno-associated virus (AAV) serotype HSC15 (rAAVHSC15) encoding human iduronate-2-sulfatase (hIDS) Recombinant human aspartylglucosaminidase Recombinant human dyskerin Recombinant human mesencephalic astrocyte-derived neurotrophic factor (MANF) Recombinant human rod-derived cone viability factor Recombinant lens epithelium derived growth factor 1-326 Recombinant microbial lipase Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2) Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor Replication-incompetent adeno-associated virus serotype 9 vector encoding the human fragile x mental retardation 1 gene Retigabine Rilonacept Rufinamide Sarizotan Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 Setmelanotide Sirolimus (Rapamycin) Small molecule agonist of 5-hydroxytryptamine serotonergic receptors Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Somatotropina ricombinante Soticlestat Stiripentol Sulindac Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin Synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts Tamoxifen citrate Temsirolimus Tenofovir disoproxil fumarate Testosterone Theranost 68 Ga RGD Timidina fosforilasi ricombinante incapsulata in eritrociti autologhi Tiratricol Trehalose Tricaprilin Trieptanoina Trofinetide Ultevursen Ursodeoxycholic acide Vatiquinone Vettore basato sulla struttura di un virus adeno-associato sierotipo 2, pseudo-sierotipizzato con un capside di tipo 8, contenente la sequenza codificante del gene umano TYMP controllato dal promotore umano di globulina che lega la tiroxina Vettore lentivirale contenente il gene dell'anemia di Fanconi A (FANCA) Vettore virale adeno-associato contenente DNA codificante un RNAi che silenzia il gene della Rodopsina/vettore virale adeno-associato contenente un gene della rodopsina Vettore virale adeno-associato del serotipo 8 contenente il gene umano GUCY2D Vettore virale adeno-associato di serotipo 8 contenente il gene umano AIPL1 Vettore virale adeno-associato di sierotipo 9 che contiene il gene della sulfamidasi umana Vettore virale adenoassociato di sierotipo 9 recante il gene dell N-acetilglucosaminidasi alpha umana Vettore virale adenoassociato recante il gene dell'alfa-N-acetilglucosaminidasi umana Vettore virale adenovirus-associato del serotipo 2 contenente il gene umano RPE65 Vettore virale adenovirus-associato del serotipo 5 contenente il gene umano pde6beta Vettore virale associato ad adenovirus di sierotipo 10 che trasporta il cDNA della N-sulfoglucosammina sulfoidrolasi umane e del fattore di modificazione 1 della sulfatasi Vettori virali associati all'adenovirus contenenti la versione modificata del gene U1-snRNA Vigabatrin Viltolarsen [5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride adeno-associated viral vector serotype 9 containing the human HGSNAT gene adenovirus associated viral vector serotype 5 containing the RPGR gene alpha-Mannosidasi ricombinate umana autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA cyclo-L-glycyl-L-2-allylproline deflazacort mRNA encoding modified human ornithine transcarbamylase polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase rimeporide rsATP7A cDNA scAAV9/JeT-GAN tetracosactide
