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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
2374
Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured
EU/3/20/2344
Treatment of acute lymphoblastic leukaemia
2373
Retifanlimab
EU/3/20/2343
Treatment of anal cancer
2372
Highly branched poly (beta-amino ester) complexed with a nanoplasmid containing the human COL7A1 gene
EU/3/20/2342
Treatment of epidermolysis bullosa
2371
Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha
EU/3/20/2341
Treatment of Fabry disease
2370
2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine
EU/3/20/2340
Diagnosis of corticobasal degeneration
2369
DNA plasmid encoding human transferrin gene
EU/3/20/2364
Treatment of retinitis pigmentosa
2368
3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v)
EU/3/20/2363
Treatment of primary ciliary dyskinesia
2367
Decitabine
EU/3/20/2338
Treatment of sickle cell disease
2366
Autologous bone marrow derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene
EU/3/20/2362
Treatment of X-linked severe combined immunodeficiency
2365
Perflubron
EU/3/20/2361
Treatment of congenital pulmonary hypoplasia
2364
(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))
EU/3/20/2336
Treatment of primary IgA nephropathy
2363
Triheptanoin
EU/3/20/2360
Treatment of carnitine palmitoyltransferase I deficiency
2362
Adeno-associated viral vector serotype 9 expressing codon-optimized human GRN gene
EU/3/20/2359
Treatment of frontotemporal dementia
2361
L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide
EU/3/20/2358
Treatment of amyotrophic lateral sclerosis
2360
Tislelizumab
EU/3/20/2357
Treatment of oesophageal cancer
2359
Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene
EU/3/20/2356
Treatment of sickle cell disease
2358
(S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one
EU/3/20/2335
Treatment of sickle cell disease
2357
Anti-(pancreatic adenocarcinoma upregulated factor) IgG1 humanised monoclonal antibody
EU/3/20/2355
Treatment of pancreatic cancer
2356
Alisitol, retinol palmitate, zinc gluconate
EU/3/20/2354
Treatment of microvillus inclusion disease
2355
Zanidatamab
EU/3/20/2353
Treatment of gastric cancer

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