1964 results
Medicine
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Orphan designation status
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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
Date of designation: 16/11/2020, Positive, Last updated: 07/03/2023 -
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Orphan designation: apomorphine hydrochloride for: Treatment of off-periods in Parkinson's disease not responding to oral treatment
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 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of designation: 16/12/2019, Positive, Last updated: 24/02/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues for: Treatment of primary hyperoxaluria
Date of designation: 31/07/2018, Positive, Last updated: 24/02/2023 -
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Orphan designation: Patidegib for: Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome)
Date of designation: 21/03/2018, Positive, Last updated: 24/02/2023 -
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Orphan designation: Recombinant human ADAMTS-13 for: Treatment of thrombotic thrombocytopenic purpura
Date of designation: 03/12/2008, Positive, Last updated: 24/02/2023 -
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Orphan designation: Omigapil maleate for: Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency
Date of designation: 08/05/2008, Positive, Last updated: 15/02/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 14/04/2004, Positive, Last updated: 13/02/2023 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of systemic sclerosis
Date of designation: 15/05/2009, Positive, Last updated: 13/02/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/03/2022, Positive, Last updated: 13/02/2023 -
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Orphan designation: Ralinepag for: Treatment of pulmonary arterial hypertension
Date of designation: 11/01/2019, Positive, Last updated: 13/02/2023 -
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Orphan designation: Rebastinib for: Treatment of ovarian cancer
Date of designation: 12/11/2021, Positive, Last updated: 07/02/2023 -
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Orphan designation: Dodecyl creatine ester, dodecyl creatine ester hydrochloride for: Treatment of creatine deficiency syndromes
Date of designation: 19/02/2021, Positive, Last updated: 06/02/2023 -
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Orphan designation: polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) for: Treatment of homocystinuria
Date of designation: 30/05/2016, Positive, Last updated: 06/02/2023 -
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Orphan designation: Volanesorsen sodium for: Treatment of familial partial lipodystrophy
Date of designation: 14/07/2016, Positive, Last updated: 02/02/2023 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
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Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) for: Treatment of post-transplantation lymphoproliferative disorders
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
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Orphan designation: Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel) for: Treatment of epidermolysis bullosa
Date of designation: 27/02/2017, Positive, Last updated: 30/01/2023 -
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Orphan designation: Bardoxolone methyl for: Treatment of autosomal dominant polycystic kidney disease
Date of designation: 20/08/2021, Positive, Last updated: 30/01/2023 -
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Orphan designation: Omaveloxolone for: Treatment of Friedreich’s ataxia
Date of designation: 27/06/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Rilonacept for: Treatment of idiopathic pericarditis
Date of designation: 06/01/2021, Positive, Last updated: 30/01/2023 -
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Orphan designation: propagermanium for: Treatment of focal segmental glomerulosclerosis
Date of designation: 19/11/2018, Positive, Last updated: 25/01/2023 -
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Orphan designation: Recombinant human mesencephalic astrocyte-derived neurotrophic factor for: Treatment of retinitis pigmentosa
Date of designation: 24/04/2015, Positive, Last updated: 24/01/2023