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活性物质： 欧盟产品号： 疾病/条件：
 欧盟EMA药品数据库_孤儿药2394个记录(本栏目收费,不能显示细节,电话13136136841)
欧盟EMA药品数据库_孤儿药
序号
活性物质
欧盟产品号
疾病/条件
2234
arimoclomol citrate
EU/3/14/1376
Treatment of Niemann-Pick's disease, type C
2233
Arimoclomol
EU/3/06/406
Treatment of amyotrophic lateral sclerosis
2232
arimoclomol citrate
EU/3/16/1659
Treatment of inclusion body myositis
2231
N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor)
EU/3/08/556
Treatment of cystic fibrosis
2230
Humanised monoclonal antibody against myostatin (domagrozumab)
EU/3/13/1105
Treatment of Duchenne muscular dystrophy
2229
Mepolizumab
EU/3/13/1116
Treatment of Churg-Strauss syndrome
2228
Mepolizumab
EU/3/04/213
Treatment of hypereosinophilic syndrome
2227
Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene
EU/3/20/2250
Treatment of Duchenne muscular dystrophy
2226
6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate
EU/3/18/2072
Treatment of cystic fibrosis
2225
hydrocortisone
EU/3/07/441
Treatment of adrenal insufficiency
2224
Magrolimab
EU/3/20/2288
Treatment of myelodysplastic syndromes
2223
Enzastaurin hydrochloride
EU/3/07/442
Treatment of diffuse large B-cell lymphoma
2222
Enzastaurin hydrochloride
EU/3/05/343
Treatment of glioma
2221
somatropin
EU/3/00/001
AIDS wasting
2220
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan)
EU/3/14/1373
Treatment of granulomatosis with polyangiitis
2219
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide
EU/3/14/1372
Treatment of microscopic polyangiitis (avacopan)
2218
1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib)
EU/3/19/2146
Treatment of biliary tract cancer
2217
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one
EU/3/14/1386
Treatment of ovarian cancer
2216
2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one
EU/3/19/2180
Treatment of myelodysplastic syndromes
2215
2-Methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one
EU/3/10/742
Treatment of acute myeloid leukaemia

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