Search for an orphan drug
61 Result(s)
- (+)-Epicatechin
- 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA
- 2'-O-methyl-phosphorothioate oligonucleotide
- 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine
- 20- hydroxyecdysone
- 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid
- 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
- 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
- Adeno-associated viral vector containing a modified U7-snRNA gene
- Adeno-associated viral vector containing modified U1 snRNA
- Adeno-associated viral vector serotype 8 containing the human MD1 gene
- Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
- Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene
- Angiotensin (1-7)
- Ataluren
- Cosyntropin
- Elamipretide HCl
- 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 52 specific phosphorothioate oligonucleotide
- Exon 53 specific phosphorothioate oligonucleotide
- Exon 55 specific phosphorothioate oligonucleotide
- Fab drug targeting the human TfR1 and conjugated to a PMO designed to bind to DMD exon 51
- Fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNA
- Givinostat
- Golodirsen
- Halofuginone hydrobromide
- Human induced pluripotent stem cell derived myogenic progenitor cells (GIVI-MPC)
- Humanised monoclonal antibody against myostatin
- Insulin-like Growth Factor-1
- 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
- Prednisone
- R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid 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
- Testosterone
- Vamorolone
- Viltolarsen
- deflazacort
- idebenone
- rimeporide
