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254 results
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Orphan designation: Human heterologous liver cells for: Treatment of ornithine-transcarbamylase deficiency
Date of designation: 14/09/2007, Positive, Last updated: 16/08/2016 -
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Orphan designation: Rubitecan for: Treatment of pancreatic cancer
Date of designation: 10/06/2003, Positive, Last updated: 23/06/2003 -
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Orphan designation: (E)-(1S,4S,10S,21R)-7-[(Z)-ethylidene]-4,21-diisopropyl-2-oxa-12,13-dithia-5,8,20,23- tetraazabicyclo[8.7.6]tricos-16-ene-3,6,9,19,22-pentone for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of designation: 28/10/2005, Positive, Last updated: 12/10/2018 -
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Orphan designation: Pralatrexate for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of designation: 13/04/2007, Positive, Last updated: 07/04/2011 -
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Orphan designation: Sodium benzoate for: Treatment of citrullinaemia type 1
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: Sodium benzoate for: Treatment of argininosuccinic aciduria
Date of designation: 18/11/2016, Positive, Last updated: 14/12/2016 -
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Orphan designation: Deferiprone for: Treatment of neurodegeneration with brain iron accumulation
Date of designation: 27/06/2018, Positive, Last updated: 04/02/2020 -
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Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N dimethylaminophosphonamidate (Eteplirsen) for: Treatment of Duchenne muscular dystrophy
Date of designation: 03/12/2008, Positive, Last updated: 22/01/2019 -
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Orphan designation: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of designation: 24/01/2013, Positive, Last updated: 29/04/2019 -
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Orphan designation: Human heterologous liver cells for: Treatment of citrullinaemia type 1
Date of designation: 08/10/2009, Positive, Last updated: 16/08/2016 -
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Orphan designation: Sodium benzoate for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: Masitinib mesilate for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole for: Treatment of mastocytosis
Date of designation: 16/11/2004, Positive, Last updated: 27/10/2005 -
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Orphan designation: Alpha-1 antitrypsin for: Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency
Date of designation: 16/11/2004, Positive, Last updated: 07/05/2019 -
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Orphan designation: Benzoic acid, sodium salt for: Treatment of non-ketotic hyperglycinaemia
Date of designation: 11/09/2002, Positive, Last updated: 08/01/2003 -
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Orphan designation: Sodium benzoate for: Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Date of designation: 18/11/2016, Positive, Last updated: 14/12/2016 -
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Orphan designation: (-)-17(Cyclopropylmethyl)-1,14 beta-dihydroxy-4,5 alpha-epoxy-6beta-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (nalfurafine) for: Treatment of uraemic pruritus
Date of designation: 11/09/2002, Positive, Last updated: 14/02/2017 -
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Orphan designation: Beclomethasone 17, 21-dipropionate for: Treatment of intestinal graft-versus-host disease
Date of designation: 13/03/2002, Positive, Last updated: 08/06/2020 -
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Orphan designation: Autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin for: Treatment of follicular lymphoma
Date of designation: 28/08/2006, Positive, Last updated: 14/06/2012 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/06/2015, Positive, Last updated: 21/05/2019 -
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Orphan designation: Murine monoclonal antibody against CD26 for: Treatment of graft-versus-host disease
Date of designation: 26/11/2010, Positive, Last updated: 22/01/2014 -
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Orphan designation: Human heterologous liver cells for: Treatment of hyperargininaemia
Date of designation: 17/12/2010, Positive, Last updated: 16/08/2016 -
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Orphan designation: Adenoviral vector containing human p53 gene for: Treatment of Li Fraumeni Syndrome
Date of designation: 23/10/2006, Positive, Last updated: 02/04/2009 -
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Orphan designation: Sodium benzoate for: Treatment of ornithine translocase deficiency
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: 27000 for: Treatment of post-polycythaemia vera myelofibrosis
Date of designation: 25/08/2010, Positive, Last updated: 29/03/2016