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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
2334
(1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride
EU/3/12/953
Treatment of West syndrome
2333
Rovalpituzumab tesirine
EU/3/16/1667
Treatment of small cell lung cancer
2332
Oxalobacter formigenes strain HC-1
EU/3/06/354
Treatment of primary hyperoxaluria
2331
Oxalobacter formigenes strain HC-1
EU/3/14/1346
Treatment of short bowel syndrome
2330
Somapacitan
EU/3/18/2068
Treatment of growth hormone deficiency
2329
pemigatinib
EU/3/19/2216
Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK
2328
Clofazimine
EU/3/19/2200
Treatment of nontuberculous mycobacterial lung disease
2327
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 (KTE-X19)
EU/3/19/2220
Treatment of mantle cell lymphoma
2326
recombinant human interleukin-3 truncated diphtheria toxin fusion protein
EU/3/15/1567
Treatment of blastic plasmacytoid dendritic cell neoplasm
2325
Oxytocin
EU/3/14/1302
Treatment of Prader-Willi syndrome
2324
dimethyl fumarate
EU/3/18/1990
Adjunctive treatment in haematopoietic cell transplantation
2323
rasagiline
EU/3/19/2174
Treatment of Duchenne muscular dystrophy
2322
Tetrahydrobiopterin
EU/3/04/199
Treatment of hyperphenylalaninaemia
2321
Fenfluramine hydrochloride
EU/3/13/1219
Treatment of Dravet syndrome
2320
adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec)
EU/3/18/2060
Treatment of spinal muscular atrophy
2319
Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA
EU/3/07/446
treatment of metachromatic leukodystrophy
2318
Miransertib
EU/3/18/1997
Treatment of Proteus syndrome
2317
Obeticholic acid
EU/3/13/1228
Treatment of primary sclerosing cholangitis
2316
Ibutamoren mesilate
EU/3/17/1882
Treatment of growth hormone deficiency
2315
Bardoxolone methyl
EU/3/18/2019
Treatment of Alport syndrome

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