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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
2294
Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene
EU/3/17/1928
Treatment of Leber's congenital amaurosis
2293
Recombinant human bone morphogenetic protein 4
EU/3/14/1348
Treatment of glioma
2292
Obiltoxaximab
EU/3/18/2065
Treatment of anthrax
2291
Picropodophyllin
EU/3/17/1904
Treatment of glioma
2290
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran)
EU/3/16/1637
Treatment of primary hyperoxaluria
2289
Dextran sulfate low molecular weight
EU/3/20/2318
Treatment of amyotrophic lateral sclerosis
2288
Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
EU/3/20/2319
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
2287
Copper histidinate
EU/3/20/2320
Treatment of Menkes disease
2286
Adeno-associated viral vector serotype 3B encoding shortened human ATP7B
EU/3/20/2321
Treatment of Wilson's disease
2285
6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one
EU/3/20/2322
Treatment of sickle cell disease
2284
Trehalose
EU/3/20/2323
Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)
2283
3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile
EU/3/20/2324
Treatment of von Hippel-Lindau disease
2282
Venglustat
EU/3/20/2325
Treatment of GM2 gangliosidosis
2281
Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta
EU/3/20/2326
Treatment of Gaucher disease
2280
Autologous T cells transduced with lentiviral vector containing a tandem chimeric antigen receptor directed against CD20 and CD19
EU/3/20/2327
Treatment of diffuse large B-cell lymphoma
2279
Human frataxin fused to TAT cell-penetrating peptide
EU/3/20/2328
Treatment of Friedreich’s ataxia
2278
Infigratinib
EU/3/20/2329
Treatment of cholangiocarcinoma
2277
Allogeneic T-cell precursors, mobilised peripheral blood-derived, ex vivo cultured
EU/3/20/2317
Treatment in haematopoietic stem cell transplantation
2276
Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli
EU/3/20/2316
Prevention of haemolytic uraemic syndrome
2275
pentosan polysulfate sodium
EU/3/20/2315
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

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