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2822 results
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Orphan designation: Govorestat for: Treatment of galactosaemia (updated)
Date of designation: 21/06/2022, Positive, Last updated: 29/03/2023 -
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Orphan designation: Iodine (131I) iobenguane for: Treatment of neuroblastoma (updated)
Date of designation: 31/01/2008, Positive, Last updated: 29/03/2023 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis (updated)
Date of designation: 16/12/2014, Positive, Last updated: 27/03/2023 -
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Orphan designation: Recombinant adeno-associated viral vector containing the human RPGR gene for: Treatment of retinitis pigmentosa caused by mutations in the RPGR gene (updated)
Date of designation: 30/05/2016, Positive, Last updated: 27/03/2023 -
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Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of maternally-inherited diabetes and deafness (updated)
Date of designation: 28/06/2019, Positive, Last updated: 27/03/2023 -
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Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (updated)
Date of designation: 10/08/2015, Positive, Last updated: 27/03/2023 -
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Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of Leigh syndrome (updated)
Date of designation: 15/10/2014, Positive, Last updated: 27/03/2023 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) for: Treatment of Wiskott-Aldrich syndrome (updated)
Date of designation: 06/06/2012, Positive, Last updated: 27/03/2023 -
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Orphan designation: Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase for: Treatment of ornithine transcarbamylase deficiency (updated)
Date of designation: 20/03/2017, Positive, Last updated: 27/03/2023 -
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Orphan designation: Soluble recombinant human fibroblast growth factor receptor 3 for: Treatment of achondroplasia (updated)
Date of designation: 27/02/2017, Withdrawn, Last updated: 24/03/2023 -
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Orphan designation: miglustat for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of designation: 11/01/2019, Withdrawn, Last updated: 24/03/2023 -
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 29/04/2009, Positive, Last updated: 24/03/2023 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 29/07/2014, Positive, Last updated: 21/03/2023 -
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Orphan designation: Autologous T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against CD7 for: Treatment of acute lymphoblastic leukaemia (updated)
Date of designation: 09/12/2022, Positive, Last updated: 14/03/2023 -
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Orphan designation: Panobinostat for: Treatment of multiple myeloma (updated)
Date of designation: 08/11/2012, Positive, Last updated: 13/03/2023 -
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Orphan designation: Lentiviral vector containing the human MYO7A gene for: Treatment of retinitis pigmentosa in Usher syndrome 1B (updated)
Date of designation: 23/03/2010, Positive, Last updated: 13/03/2023 -
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Orphan designation: Lentiviral vector containing the human ABCA4 gene for: Treatment of Stargardt's disease (updated)
Date of designation: 02/02/2010, Positive, Last updated: 13/03/2023 -
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Orphan designation: clofazimine for: Treatment of nontuberculous mycobacterial lung disease (updated)
Date of designation: 21/08/2019, Positive, Last updated: 13/03/2023 -
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Orphan designation: 5-aminolevulinic acid for: Treatment of glioma (updated)
Date of designation: 12/01/2017, Positive, Last updated: 10/03/2023 -
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Orphan designation: Cannabidiol for: Treatment of 22q11.2 deletion syndrome (updated)
Date of designation: 11/11/2022, Positive, Last updated: 07/03/2023 -
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Orphan designation: Cannabidiol for: Treatment of fragile X syndrome (FXS) (updated)
Date of designation: 25/02/2022, Positive, Last updated: 07/03/2023 -
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Orphan designation: 3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v) for: Treatment of primary ciliary dyskinesia (updated)
Date of designation: 16/11/2020, Positive, Last updated: 07/03/2023 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C (updated)
Date of designation: 19/11/2014, Positive, Last updated: 01/03/2023 -
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Orphan designation: apomorphine hydrochloride for: Treatment of off-periods in Parkinson's disease not responding to oral treatment (updated)
Date of designation: 16/02/2006, Positive, Last updated: 28/02/2023 -
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Orphan designation: Ribitol for: Treatment of limb-girdle muscular dystrophy
Date of designation: 19/10/2020, Positive, Last updated: 27/02/2023