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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
2314
Antisense oligonucleotide targeting the F508delta mutation of CFTR (eluforsen)
EU/3/13/1195
Treatment of cystic fibrosis
2313
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor)
EU/3/14/1361
Treatment of systemic sclerosis
2312
5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]
EU/3/17/1916
Treatment of biliary tract cancer
2311
Recombinant antibody derivative against human CD19 and CD3 (blinatumomab)
EU/3/03/176
Treatment of chronic lymphocytic leukaemia
2310
Recombinant antibody derivative against human CD19 and CD3 (blinatumomab)
EU/3/03/175
Treatment of mantle cell lymphoma
2309
Treosulfan
EU/3/04/186
Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
2308
Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec)
EU/3/15/1509
Treatment of spinal muscular atrophy
2307
pentetrazol
EU/3/15/1569
Treatment of idiopathic hypersomnia
2306
copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin
EU/3/13/1138
Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
2305
Ecothiopate iodide
EU/3/15/1474
Treatment of Stargardt's disease
2304
Allantoin
EU/3/13/1232
Treatment of epidermolysis bullosa
2303
(S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid)
EU/3/07/513
Treatment of tuberculosis
2302
Alpha-1 proteinase inhibitor
EU/3/07/474
Treatment of cystic fibrosis
2301
Sodium cromoglicate
EU/3/20/2294
Treatment of idiopathic pulmonary fibrosis
2300
Adeno-associated viral vector expressing acid alpha-glucosidase gene
EU/3/20/2303
Treatment of glycogen storage disease type II (Pompe's disease)
2299
5-aminolevulinic acid
EU/3/16/1811
Treatment of glioma
2298
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
EU/3/12/1098
Treatment of retinitis pigmentosa
2297
L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin
EU/3/11/923
Treatment of glioma
2296
Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
EU/3/17/1898
Treatment of Wilson's disease
2295
Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine)
EU/3/15/1597
Treatment of acute myeloid leukaemia

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