欧盟EMA药品数据库_孤儿药_药药网

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

2354

(R)‑tetrahydrofuran‑3‑yl 4‑(6‑(5‑(4‑ethoxy‑1‑isopropylpiperidin‑4‑yl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate

Treatment of fibrodysplasia ossificans progressiva

2353

Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene

Treatment of aspartylglucosaminuria

2352

Treatment of peripheral T-cell lymphoma

2351

Treatment of KCNQ2 developmental and epileptic encephalopathy

2350

Treatment of multiple myeloma

2349

5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid)

Treatment of Duchenne muscular dystrophy

2348

1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt

Treatment of polycythaemia vera

2347

human anti-promyostatin monoclonal antibody (apitegromab)

Treatment of spinal muscular atrophy

2346

Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel)

Treatment of diffuse large B-cell lymphoma

2345

Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel)

Treatment of follicular lymphoma

2344

Lisocabtagene maraleucel

Treatment of primary mediastinal large B-cell lymphoma

2343

Human monoclonal antibody against Pseudomonas aeruginosa IATS-O1

Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa

2342

RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride

Treatment of Duchenne muscular dystrophy

2341

Sodium thiosulfate

Treatment of calciphylaxis

2340

Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)

Treatment of Crigler-Najjar syndrome

2339

Heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate)

Treatment of traumatic spinal cord injury

2338

Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (pegcetacoplan)

Treatment of C3 glomerulopathy

2337

2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione

Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes

2336

Treatment of moderate and severe closed traumatic brain injury

2335

2-Hydroxyoleic acid

Treatment of glioma

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