Orphanet: Search for an orphan drug
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50 Resultaat/Resultaten
(+)-Epicatechin 17-alfa,21-dihydroxy-16-alfa-methylpregna-1,4,9(11)-trieen-3,20-dion 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine 20- hydroxyecdysone 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole Adeno-associated viral vector containing a modified U7-snRNA gene Adeno-associated viral vector containing modified U1 snRNA Adeno-associated viral vector containing the human alpha-sarcoglycan gene Adeno-associated viral vector containing the human gamma-sarcoglycan gene Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene Asp-Arg-Val-Tyr-Ile-His-Pro Ataluren Eteplirsen 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 44 specific phosphorothioate oligonucleotide Exon 45 specific phosphorothioate oligonucleotide Exon 51 specific phosphorothioate oligonucleotide Exon 53 specific phosphorothioate oligonucleotide Exon 55 specific phosphorothioate oligonucleotide Exon 55 specifiek phosphorothioate oligonucleotide Gehumaniseerd anti-myostatine monoklonaal antilichaam Givinostat Golodirsen Halofuginone Hydrobromide Idebenone Kifunensine Metformin and L-citrulline N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide Naproxcinod Pamrevlumab R, S-O-(3-piperidino-2-hydroxy-1-propyl) - nicotinezuur amidoxime dihydrochloride 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 adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Recombinant fusion protein consisting of the extracellular portion of human activin receptor IIB linked to the human IgG1 Fc domain Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA Tamoxifen citrate Viltolarsen deflazacort drisapersen rimeporide tetracosactide
