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2661 results
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Orphan designation: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for: Treatment of Fanconi anaemia type A (updated)
Date of designation: 17/12/2010, Positive, Last updated: 11/11/2022 -
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Orphan designation: afatinib for: Treatment of Fanconi anaemia (updated)
Date of designation: 14/12/2018, Positive, Last updated: 11/11/2022 -
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Orphan designation: dimethyl fumarate for: Treatment of adrenoleukodystrophy (updated)
Date of designation: 09/01/2020, Positive, Last updated: 11/11/2022 -
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Orphan designation: Autologous skin equivalent graft composed of keratinocytes and fibroblasts genetically corrected by CRISPR/Cas9-mediated excision of mutation-carrying COL7A1 exon 80 for: Treatment of epidermolysis bullosa (updated)
Date of designation: 28/02/2020, Positive, Last updated: 11/11/2022 -
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Orphan designation: Adeno-associated virus of serotype rh10 encoding Human MLC1 under the control of GFAP promoter for: Treatment of megalencephalic leukoencephalopathy with subcortical cysts (updated)
Date of designation: 15/10/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Autologous CD34+ enriched cells transduced with a self-inactivating lentiviral vector containing the codon-optimized RPS19 gene for: Treatment of Diamond-Blackfan anaemia (updated)
Date of designation: 12/11/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Fingolimod for: Treatment of adrenoleukodystrophy (updated)
Date of designation: 12/11/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR gene for: Treatment of pyruvate kinase deficiency (updated)
Date of designation: 24/02/2022, Positive, Last updated: 11/11/2022 -
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Orphan designation: erlotinib for: Treatment of patients with Olmsted syndrome (updated)
Date of designation: 09/12/2020, Positive, Last updated: 11/11/2022 -
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis (updated)
Date of designation: 18/12/2002, Positive, Last updated: 11/11/2022 -
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Orphan designation: genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII (beremagene geperpavec) for: Treatment of epidermolysis bullosa (updated)
Date of designation: 16/04/2018, Positive, Last updated: 10/11/2022 -
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Orphan designation: Burosumab for: Treatment of phosphaturic mesenchymal tumour (updated)
Date of designation: 16/04/2018, Positive, Last updated: 08/11/2022 -
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Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one (Tovinontrine) for: Treatment of sickle cell disease (updated)
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022 -
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Orphan designation: Infigratinib for: Treatment of cholangiocarcinoma (updated)
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022 -
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Orphan designation: (2S)-2-[(4R)-2-oxo-4-propyltetrahydro-1H-pyrrol-1-yl] butanamide (Brivaracetam) for: Treatment of progressive myoclonic epilepsies (updated)
Date of designation: 26/08/2005, Withdrawn, Last updated: 08/11/2022 -
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Orphan designation: Sodium phenylbutyrate for: Treatment of pyruvate dehydrogenase complex deficiency (updated)
Date of designation: 11/11/2015, Positive, Last updated: 07/11/2022 -
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Orphan designation: Autologous haematopoietic stem and progenitor cell population containing CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA ex vivo expanded for: Treatment of osteopetrosis (updated)
Date of designation: 15/10/2021, Positive, Last updated: 07/11/2022 -
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Orphan designation: Alisporivir for: Treatment of collagen VI-related myopathies (updated)
Date of designation: 16/03/2022, Positive, Last updated: 07/11/2022 -
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Orphan designation: Tamoxifen citrate for: Treatment of neuronal ceroid lipofuscinosis (updated)
Positive, Last updated: 07/11/2022 -
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Orphan designation: Lentiviral vector encoding human coagulation factor IX for: Treatment of haemophilia B (updated)
Date of designation: 26/02/2019, Positive, Last updated: 07/11/2022 -
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Orphan designation: Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins for: Treatment of Stargardt's disease (updated)
Date of designation: 17/10/2019, Positive, Last updated: 07/11/2022 -
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Orphan designation: Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins for: Treatment of inherited retinal dystrophies (updated)
Date of designation: 28/02/2020, Positive, Last updated: 07/11/2022 -
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Orphan designation: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human ADA2 gene for: Treatment of adenosine deaminase 2 deficiency (DADA2) (updated)
Date of designation: 20/08/2021, Positive, Last updated: 07/11/2022 -
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Orphan designation: Mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene for: Treatment of Stargardt's disease (updated)
Date of designation: 04/07/2014, Positive, Last updated: 07/11/2022 -
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Orphan designation: Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for: Treatment of Usher syndrome (updated)
Date of designation: 04/07/2014, Positive, Last updated: 07/11/2022