Orphanet: Wyszukaj lek sierocy
Wyszukaj lek sierocy
175 Wyniki
- (14S)-8-[3-(2-{dispiro[2.0.24.13]heptan-7-yl}ethoxy)-1H-pyrazol-1-yl]-12,12-dimethyl-2lamba6-thia-3,9,11,18,23-penta-azatetracyclo[17.3.1.111,14.05,10]tetracosa-1(22),5,7,9,19(23),20-hexaene-2,2,4-trione calcium salt hydrate, deutivacaftor, tezacaftor
- (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid
- (R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide
- (R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate
- (S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt
- 11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione
- 2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl -(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)- 5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenine, sodium salt
- 2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanoylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-uridine sodium salt
- 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide
- 3-(6-(1-(2,2-difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3- methylpyridin-2-yl)benzoic acid
- 4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid
- 4,6,4'-trymethylangelicin
- 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid
- 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate
- 8-cyclopentyl-1,3-dipropylxanthine
- Acetylleucine
- Adeno -associated viral vector serotype 8 containing the human functional version of UGT1A1 gene
- Adeno-associated viral vector containing porphobilinogen deaminase gene
- Adeno-associated viral vector expressing acid alpha-glucosidase gene
- Adeno-associated viral vector serotype 2.5T encoding the human cystic fibrosis transmembrane conductance regulator with a partial deletion in the R domain
- Adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A
- Adeno-associated viral vector serotype 3B encoding shortened human ATP7B
- Adeno-associated viral vector serotype 9 encoding human ATP7B
- Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
- Afamelanotide
- Alginate oligosaccharide (G-block) fragment
- Alpha-1 antitrypsin (inhalation use)
- Alpha-1 proteinase inhibitor
- Alpha-1 proteinase inhibitor (inhalation use)
- Amikacin sulfate
- Amikacin sulfate (liposomal)
- Amiloride hydrochloride dihydrate
- Ammonium tetrathiomolybdate
- Antisense oligonucleotide consisting of 18 nucleotide residues with a sequence of 5'-5MeC-5MeU-5MeU-5MeU-5MeC-5MeC-5MeU-5MeU-5MeC-A-5MeC-5MeU-G-5MeU- 5MeU-G-5MeC-A-3'
- Ataluren
- Avian polyclonal IgY antibody against Pseudomonas aeruginosa
- Aztreonam lysinate (inhalation use)
- Bacterial lipase
- Belcesiran
- Bitopertin
- Carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methylethyl] phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester
- Chenodeoxycholic acid
- Cholic acid
- Choline tetrathiomolybdate
- Ciprofloxacin (inhalation use)
- Ciprofloxacin (liposomal)
- Colistimethate sodium
- Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
- Cysteamine
- Cystic fibrosis transmembrane conductance regulator (CFTR) amplifier
- Deferasirox
- Deferiprone
- Denufosol tetrasodium
- Dersimelagon
- Dexamethasone sodium phosphate encapsulated in human erythrocytes
- Dimethyl fumarate
- Dornase alfa
- Duramycin
- Elexacaftor/Tezacaftor/Ivacaftor
- Eluforsen
- Engineered protein inhibitor of human neutrophil elastase
- Fixed-dose combination of fosfomycin disodium and tobramycin
- Gallium citrate
- Givosiran
- Glutathione
- Glycerol phenylbutyrate
- Govorestat
- Heparin sodium
- Herpes simplex virus 1 expressing the human CFTR gene
- Heterologous human adult liver derived stem cells
- Heterologous human adult liver-derived progenitor cells
- Human alpha 1-antitrypsin
- Human alpha1-proteinase inhibitor (respiratory use)
- Hypothiocyanite / lactoferrin
- Ivacaftor
- L-Lysine-N-acetyl-L-cysteinate
- Lenabasum
- Liprotamase
- Lumacaftor / Ivacaftor
- Mannitolum
- Maralixibat chloride
- Menadione sodium bisulfite
- Methyl (R)-4-(3S,5R,7R,8R,9S,10S,13R,14S,17R)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-2-yl-methyl)amino)butyl)amino) hexadecahydro-1H-cyclopenta[a]phenanthren-17-yl) pentan
- Modified mRNA encoding the UGT1A1 protein
- Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol
- N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene
- Nafamostat mesilate
- Nitisinone
- Nitric oxide
- Octovalent Pseudomonas aeruginosa O-polysaccharie-toxin A conjugate vaccine
- Olipudase alfa
- Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab' antibody
- Phosphoinositide 3-kinase gamma peptide
- Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent
- Recombinant adeno-associated viral vector containing human alpha-1 antitrypsin gene
- Recombinant dog gastric lipase
- Recombinant human CXCL8 mutant
- Recombinant human acid ceramidase
- Recombinant human alpha-1-antitrypsin (respiratory use)
- Recombinant human bile salt-stimulated lipase
- Recombinant human porphobilinogen deaminase
- Recombinant microbial lipase
- Sebelipase alfa
- Sinapultide, dipalmitoylphosphatidylcholine, palmitoyloleoylphosphatidylglycerol and palmitic acid
- Sodium ascorbate and menadione sodium bisulfite
- Synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol-conjugated, acyclic nucleobase analogue
- Tamoxifen citrate
- Teicoplanin
- Temocillin sodium
- Tezacaftor
- Tezacaftor / Ivacaftor
- Titanium dioxide and bisoctrizole
- Tobramycin (inhalation powder)
- Tobramycin (inhalation solution)
- Tobramycin (inhalation use)
- Tobramycin (liposomal)
- Trientine dihydrochloride
- Ursodeoxycholic acid
- Vatiquinone
- Volrubigene ralaparvovec
- Zinc acetate
- adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene
- adeno-associated viral vector serotype 8 encoding the human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
- codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles
- ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor
- levofloxacin hemihydrate
- mRNA encoding human glucose-6-phosphatase variant S298C
- mRNA encoding the human glycogen debranching enzyme
- poly-N (acetyl, arginyl) glucosamine (PAAG)
- sodium nitrite and EDTA
- trientine tetrahydrochloride
