Orphanet: Wyszukaj lek sierocy
Wyszukaj lek sierocy
180 Wyniki
- ((S)-3-(1-Cyclopentyl-5-(2-(trifluoromethyl)phenyl)-1H-pyrazole-3-carboxamido)-5-(3,3-difluoropiperidin-1-yl)pentanoic acid hydrochloride)
- (1'R,6'R)-3-(benzylamine)-6-hydroxy-3'-methyl-4-pentyl-6'-(prop-1-en-2-yl)-[1,1'-bi(cyclohexane)]-2',3,6-triene-2,5-dione
- (S)-N-(5-(1-(3-ethoxy-4-methoxyphenyl)-2-(methylsulfonyl)ethyl)-4,6-dioxo-5,6-dihydro-4H-thieno[3,4-c]pyrrol-1-yl)acetamide
- 1,1'-(1, 12- Dodecanediyl)bis[cyclopropanecarboxamide]
- 1-[12-(1 Carboxycyclopropyl) dodecyl] cyclopropane-1-carboxylic acid
- 2-((2-fluoro-4-iodophenyl)amino)-N-(2-hydroxyethoxy)-1-methyl-1H-pyrrolo[2,3-b]pyridine-3-carboxamide
- 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-c]pyridine-3,6(2H,5H)-dione
- 2-[1-(3-{6-[(1E)-(hydroxyimino)methyl]-5-methyl-4-oxo-7-propyl-3H,4H-pyrrolo[2,1-f][1,2,4]triazin-2-yl}-4-propoxybenzenesulfonyl)piperidin-4-yl]ethyl nitrate
- 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid
- 2-{2,6-Dichloro-3-[(2,2-dimethyl-propionylamino)-methyl]-phenylamino}-6-(2,2-difluoroethoxy)-1-methyl-1H-benzoimidazole-5-carboxylic acid (trans-4-trifluoromethylcyclohexyl)-amide, hydrogensulfate
- 3-Pentylbenzeneacetic acid sodium salt
- 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide
- 5-Arylnicotinamide ABCA1 inducer
- Abetimus sodium
- Adalimumab
- Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells
- Allogeneic aortic endothelial cells cultured in a porcine gelatin matrix
- Antagonist of the complement 5a receptor
- Anti-von Willebrand Aptamer
- Apadamtase alfa
- Apremilast
- Autologous adipose tissue-derived mesenchymal stem cells
- Autologous bone marrow-derived mononuclear cell fraction
- Avacopan
- Aviptadil
- Bardoxolone methyl
- Begelomab
- Belimumab
- Birtamimab
- Bosentan
- Brodalumab
- Canakinumab
- Cannabidiol
- Caplacizumab
- Caprine hyperimmune serum against HIV lysate
- Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate
- Chemically modified full phosphorothioate-backbone anti-miR-17 oligonucleotide
- Chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4
- Chimeric human-murine IgG1 kappa monoclonal antibody against TNF alfa
- Colchicine
- Complement factor H
- Corticotropin (ACTH) porcine (synthetic)
- Cudetaxestat
- Danicopan
- Daratumumab
- Defibrotide
- Eculizumab
- Efzofitimod
- Eprodisate
- Etidronate disodium
- Everolimus
- Felzartamab
- Fixed-dose combination of potassium citrate and potassium bicarbonate
- Fresolimumab
- Fusion protein composed of the first 2 immunoglobulin-like domains of the human roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment
- Ganaxolone
- Gevokizumab
- Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein
- Granulocyte-macrophage colony-stimulating factor
- Gusperimus trihydrochloride
- Human IgG1Fc- iMod domain (amino acids 2-60) of human HARS fusion protein
- Human engineered monoclonal antibody specific for transforming growth factor ß1
- Human normal immunoglobulin
- Humanized IgG1 monoclonal antibody binding to CD19
- Immune Globulin (Human)
- Inaxaplin
- Iodine (123I) Serum Amyloid P
- Iodine (124I) evuzamitide
- Iptacopan
- Ixazomib
- L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser
- L-cystine bis(N-methylpiperazide)
- L-ergothioneine
- Lademirsen
- Lanifibranor
- Lanreotide acetate
- Lenabasum
- Levamisol hydrochloride
- Lixivaptan
- Mebendazole
- Menadione sodium bisulfite
- Mepolizumab
- Mirdametinib
- N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide
- Narsoplim
- Nintedanib
- Nitric oxide
- Nitroglycerin
- Oxypurinol
- PEGylated peptide inhibitor of complement C3
- Paquinimod
- Pegcetacoplan
- Peptide 144 TGF-beta1-inhibitor (TSLDASIIWAMMQN)
- Phage-derived, non-replicative delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli (STEC)
- Pioglitazone
- Pomalidomide
- Propagermanium
- Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli
- Recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1
- Recombinant human minibody against complement component C5
- Recombinant human residue 41 glutamic acid to glutamine variant of Interferon-alfa-2b
- Recombinant human type I pancreatic elastase
- Recombinant humanised anti-human interleukin-1 beta monoclonal antibody
- Recombinant humanized anti-transforming growth factor (TGF) beta 3 antibody
- Recombinant monoclonal antibody to human serum amyloid P component
- Rilonacept
- Riociguat
- Rituximab
- Romilkimab
- S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)
- Selumetinib
- Setmelanotide
- Single stranded, chemically modified oligonucleotide that binds to and inhibits the function of micro RNA-21
- Siponimod
- Sirolimus
- Sodium thiosulfate
- Solnatide
- Sparsentan
- Synthetic double-strand small interfering ribonucleic acid (siRNA) oligonucleotide drug directed against C3 messenger RNA
- Terguride
- Tiopronine
- Tocilizumab
- Tolvaptan
- Treprostinil diethanolamine (oral use)
- Tripotassium citrate monohydrate and potassium hydrogen carbonate
- Type I native bovine skin collagen
- Umbilical cord mesenchymal stem cell secretome
- Vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar
- Venglustat
- Vonafexor
- [4-Aminobutanoic acid-glycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-L-aspartyl](cyclo 1-Dgamma17)
- autologous adipose tissue-derived stromal vascular fraction cells
- equine immunoglobulin F(ab')2 fragments targeting Shiga toxin
- florbetaben (18F)
- humanised IgG1K monoclonal antibody against interferon beta
- imlifidase
- potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo- 5-phenyl-6,7-dihydrothieno[2, 3-b]pyridin-4-olate hydrate
- ziritaxestat
