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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
2254
Hemopexin
EU/3/20/2298
Treatment of sickle cell disease
2253
Acalabrutinib
EU/3/16/1624
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
2252
Recombinant human beta-glucuronidase (vestronidase alfa)
EU/3/12/973
Treatment of mucopolysaccharidosis type VII (Sly syndrome)
2251
Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B-
EU/3/15/1599
Treatment of haemophilia B
2250
Triheptanoin
EU/3/15/1526
Treatment of carnitine palmitoyltransferase II deficiency
2249
Triheptanoin
EU/3/15/1525
Treatment of mitochondrial trifunctional protein deficiency
2248
Triheptanoin
EU/3/15/1524
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
2247
Triheptanoin
EU/3/15/1508
Treatment of very long-chain acyl-CoA dehydrogenase deficiency
2246
adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec)
EU/3/16/1771
treatment of glycogen storage disease type Ia
2245
Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec)
EU/3/16/1623
Treatment of ornithine transcarbamylase deficiency
2244
Setmelanotide
EU/3/16/1688
Treatment of Prader-Willi syndrome
2243
Setmelanotide
EU/3/16/1703
Treatment of pro-opiomelanocortin deficiency
2242
Setmelanotide
EU/3/18/2101
Treatment of leptin receptor deficiency
2241
Setmelanotide
EU/3/19/2245
Treatment of Alström syndrome
2240
Setmelanotide
EU/3/19/2192
Treatment of Bardet-Biedl syndrome
2239
humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain
EU/3/15/1575
Prevention of graft-versus-host disease
2238
(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt (niraparib)
EU/3/10/760
Treatment of ovarian cancer
2237
Amikacin sulfate
EU/3/14/1259
Treatment of nontuberculous mycobacterial lung disease
2236
Amikacin sulfate
EU/3/06/387
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
2235
5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate
EU/3/19/2228
Treatment of multiple system atrophy

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