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Orphan Designations by geographical area
- 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene
- 1,2:5,6-Dianhydrogalactitol
- 1-[2-(Benzo[1,2,5]thiadiazol-5-ylamino)-6-(2,6-dichloro-phenyl)-pyrido[2,3-d]pyrimidin-7-yl]-3- tert-butyl-urea
- 1,2-bis(methylsulphonyl)-1-(2-chloroethyl)-2-[(methylamino)carbonyl]hydrazine
- 1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene
- 1-{3-[3-(4-chlorophenyl)propoxy]propyl}piperidine, hydrochloride
- 1,3-Propanedisulfonic acid, disodium salt
- 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one
- 1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea
- 16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7-amino acid peptide
- 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide
- 17-allylamino-17-demethoxygeldanamycin
- (-)-17-(cyclopropylmethyl)-3,14 ß-dihydroxy-4,5 a-epoxy-6ß-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride
- 17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5)
- 1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea
- 1-deoxynojirimycin hydrochloride
- (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate
- (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt
- (1R,2S)6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol
- (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride
- 20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'-carboxylate
- 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-c]pyridine-3,6(2H,5H)-dione
- 2,2-dimethylbutyric acid, sodium salt
- 2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}- 1H-pyrazol-3-yl)amino]- quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate
- 2,3,4,5 tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride
- 2',3',5'-tri-O-acetyluridine
- 2-{4-[(5,6-diphenylpyrazin-2-yl)(isopropyl)amino]butoxy}-N-(methylsulfonyl)acetamide
- 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid
- 26 base single stranded phosphodiester DNA oligonucleotide
- 2-Allyl-1-[6-(1-hydroxy-1-methylethyl)pyridin-2-yl]-6-{[4-(4-methylpiperazin-1-yl)phenyl]amino}-1,2-dihydro-3H-pyrazolo[3,4-d]pyrimidin-3-one
- [2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]
- 2-hydroxyoleic acid
- 2-iminobiotin
- 2-Methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]-phenol
- 2-methoxymethyl-2-hydroxymethyl-1-azabicyclo [2,2,2] octan-3-one
- (2R)-3-(2-hydroxymethylindanyl-4-oxy)-phenyl-4,4,4-trifluorobutane-1-sulfonate
- (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
- 2S 4R ketoconazole
- 3,4-diaminopyridine phosphate
- 3-(6-(1-(2,2-difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3- methylpyridin-2-yl)benzoic acid
- 3-methoxy-pregnenolone
- (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt
- 4-[123I] iodo-L-phenylalanine
- 4-[131I]iodo-L-phenylalanine
- 4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
- 4-[3-(methylsulfonyl)phenyl]-1-propylpiperidine x HC1
- 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide
- 4,6,8-trihydroxy-10-(3,7,11-trimethyldodeca-2,6,10-trienyl)-5,10-dihydrodibenzo[b,e][1,4] diazepin-11-one
- 4,7,10,13,16,19-Docosahexaenoic acid
- 4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol
- 4-Amino-1-[5-O-[(2R,4S)-2-oxido-4-(4-pyridinyl)-1,3,2-dioxaphosphorinan-2-yl]-D-arabinofuranosyl]- 2(1H)-pyrimidinone
- 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline
- 4-amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride
- 4-aminopyridine
- 4-benzyl-2-naphtalen-1-yl-1,2,4-thiadiazolidine-3,5-dione
- 4-ethoxy-2-(piperazin-1-yl)-7-(pyridin-4-yl)-5H-pyrimido[5,4-b]indol
- 4-imino-1, 3-diazobicyclo-[3.1.0]-hexan-2-one
- 5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
- 5-(2,6-Difluoro-phenoxy)-3(R,S)-{2(S)-[2(S)-(3-methoxycarbonyl-2(S)-{3-methyl-2(S)-[(quinoline-2-carbonyl)-amino]-butyrylamino}-propionylamino)-3-methyl-butyrylamino]-propionylamino}-4-oxo-pentanoic acid methyl ester
- 5-aminolevulinic acid hydrochloride
- 5'-CTG CCA CGT TCT CCT GC-(2' methoxy)A-(2' methoxy)C-(2' methoxy)C-3'
- 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
- 5-methyl-pyridine-2-sulfonic acid {6-(2-hydroxy-ethoxy)-5-(2-methoxy-phenoxy)-2-[2-(1H-tetrazol-5-yl)-pyridin-4-yl]-pyrimidin-4-yl}-amide sodium salt
- 5'-O-(trans-9''-octadecenoyl)-1-beta-D-2'deoxy-2',2'-difluorocytidine
- 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine
- 5(S)-(2'-hydroxy ethoxy)-20(S)-camptothecin
- 6alpha-ethyl-chenodeoxycholic acid
- 6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide
- 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one
- (6R)-4, 5, 6, 7-tetrahydro-N6-propyl-2, 6-benzothiazolediamine dihydrochloride monohydrate
- 6-thioguanine (oral liquid)
- 7-beta-hydroxycholesteryl-3-beta-oleate
- 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride
- 9-cis-Retinyl acetate
