20662 results
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Efficacy Working Party (EWP)
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Scientific Advisory Group on Clinical Neuroscience
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Scientific Advisory Group on Antimicrobials
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National expert: Ioana Agache, European Medicines Agency (updated)
- Declaration of interests - 43.23 KB | PDF
- Curriculum Vitae - 189.54 KB | PDF
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Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/07/2013, Positive, -
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Orphan designation: Acetylcysteine for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 26/01/2005, Withdrawn, -
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
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Orphan designation: Plitidepsin for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 23/02/2011, Withdrawn, -
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Orphan designation: 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)- 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl -(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl... for: Treatment of diffuse large B-cell lymphoma
Date of designation: 10/08/2015, Withdrawn, -
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Orphan designation: Propranolol for: Treatment of soft tissue sarcoma
Date of designation: 12/12/2016, Positive, -
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Orphan designation: [N-((2S,3R,3aS,3'R,4a'R,6S,6a'R,6b'S,7aR,12a'S,12b'S,Z)-3,6,11',12b'-tetramethyl-2',3a,3',4,4',4a',5,5',6,6',6a',6b',7,7a,7',8',10',12',12a',12b'-icosahydro-1'H,3H-spiro[furo[3,2-b]pyridine-2,9'-naphtho[2,1-a]azulene]-3'-yl)methanesulfonamide hydrochloride] for: Treatment of chondrosarcoma
Date of designation: 13/05/2011, Withdrawn, -
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Orphan designation: Idelalisib for: Treatment of splenic marginal-zone lymphoma
Date of designation: 05/08/2013, Withdrawn, -
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Orphan designation: Repertaxin L-lysine salt for: Prevention of delayed graft function in organ transplant
Date of designation: 19/09/2001, Positive, -
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Orphan designation: Trientine tetrahydrochloride for: Treatment of Wilson's disease
Date of designation: 19/03/2015, Withdrawn, -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of designation: 15/10/2014, Positive, -
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Orphan designation: Human MHC non-restricted cytotoxic T-cell line for: Treatment of ovarian cancer
Date of designation: 30/11/2009, Positive, -
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Orphan designation: Nitric oxide for: Treatment of cystic fibrosis
Date of designation: 15/10/2014, Positive, -
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Orphan designation: Volasertib for: Treatment of acute myeloid leukaemia
Date of designation: 26/03/2014, Positive, -
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Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 08/11/2012, Withdrawn, -
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Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of designation: 11/04/2014, Positive, -
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Orphan designation: Recombinant human serum amyloid P for: Prevention of scarring post glaucoma filtration surgery
Date of designation: 09/10/2009, Withdrawn, -
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Orphan designation: Methotrexate for: Treatment of alkaptonuria
Date of designation: 29/08/2016, Positive, -
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Orphan designation: Vatreptacog alfa (activated) for: Treatment of haemophilia B
Date of designation: 09/08/2012, Withdrawn, -
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Orphan designation: Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys for: Treatment of high-altitude pulmonary oedema
Date of designation: 08/02/2013, Positive, -
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Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of designation: 31/07/2001, Positive,