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426 results
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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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Withdrawn application: Advexin
date of withdrawal: 17/12/2008, Initial authorisation, Last updated: 19/12/2009 -
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Withdrawn application: Cerepro
date of withdrawal: 13/07/2007, Initial authorisation, Last updated: 20/07/2007 -
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Withdrawn application: Bosatria
mepolizumab, date of withdrawal: 28/07/2009, Initial authorisation, Last updated: 29/07/2009 -
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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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Withdrawn application: Zafiride
date of withdrawal: 01/06/2017, Initial authorisation, Last updated: 08/02/2018 -
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Withdrawn application: Elmisol
date of withdrawal: 29/05/2017, Initial authorisation, Last updated: 18/07/2017 -
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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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Withdrawn application: Prohippur
date of withdrawal: 03/04/2018, Initial authorisation, Last updated: 05/07/2018 -
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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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Withdrawn application: Winfuran
date of withdrawal: 17/01/2014, Initial authorisation, Last updated: 11/03/2014 -
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Withdrawn application: Theraloc
date of withdrawal: 01/12/2008, Initial authorisation, Last updated: 04/12/2008 -
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Withdrawn application: Xegafri
date of withdrawal: 05/05/2016, Initial authorisation, Last updated: 22/06/2016 -
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Withdrawn application: Vekacia
ciclosporin, date of withdrawal: 14/11/2008, Initial authorisation, Last updated: 18/11/2008 -
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Withdrawn application: Graspa
date of withdrawal: 14/11/2016, Initial authorisation, Last updated: 25/04/2017 -
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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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Withdrawn application: Tyvaso
date of withdrawal: 17/02/2010, Initial authorisation, Last updated: 17/05/2010 -
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Withdrawn application: Ethyl Eicosapent soft gelatin capsules
date of withdrawal: 01/12/2009, Initial authorisation, Last updated: 08/12/2009 -
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Withdrawn application: Corluxin
date of withdrawal: 23/03/2015, Initial authorisation, Last updated: 12/06/2015