2257 results
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Orphan designation: for: Treatment of congenital alpha-1 antitrypsin deficiency (updated)
Date of first decision: 20/03/2007, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of primary sclerosing cholangitis (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of primary biliary cirrhosis (updated)
Date of first decision: 22/08/2014, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of anaplastic thyroid cancer (updated)
Date of first decision: 14/04/2004, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of gastro-entero-pancreatic neuroendocrine tumours (updated)
Date of first decision: 21/03/2016, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of glioma (updated)
Date of first decision: 19/02/2014, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of ovarian cancer (updated)
Date of first decision: 17/07/2013, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of achromatopsia caused by mutations in the CNGA3 gene (updated)
Date of first decision: 09/10/2015, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of achromatopsia caused by mutations in the CNGB3 gene (updated)
Date of first decision: 08/02/2013, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of X-linked juvenile retinoschisis (updated)
Date of first decision: 12/03/2013, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of retinitis pigmentosa caused by mutations in the RPGR gene (updated)
Date of first decision: 30/05/2016, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of fragile X syndrome (updated)
Date of first decision: 20/04/2017, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of snakebite envenomation (updated)
Date of first decision: 09/10/2015, Positive, Last updated: 19/02/2020 -
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Orphan designation: for: Treatment of chronic lymphocytic leukaemia (updated)
Date of first decision: 19/01/2009, Withdrawn, Last updated: 18/02/2020 -
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Orphan designation: for: Treatment of multiple myeloma (updated)
Date of first decision: 19/01/2009, Withdrawn, Last updated: 18/02/2020 -
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Orphan designation: for: Treatment of limb-girdle muscular dystrophy (updated)
Date of first decision: 27/02/2017, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: for: treatment of facioscapulohumeral muscular dystrophy (updated)
Date of first decision: 12/02/2015, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: for: Treatment of glioma (updated)
Date of first decision: 27/10/2011, Positive, Last updated: 12/02/2020 -
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Orphan designation: for: Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients (updated)
Date of first decision: 16/01/2014, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: for: Treatment of follicular lymphoma (updated)
Date of first decision: 24/01/2013, Withdrawn, Last updated: 11/02/2020 -
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Orphan designation: for: Treatment of pulmonary arterial hypertension (updated)
Date of first decision: 10/07/2008, Withdrawn, Last updated: 11/02/2020 -
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Orphan designation: for: Treatment of pseudomyxoma peritonei (updated)
Date of first decision: 14/12/2018, Positive, Last updated: 05/02/2020 -
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Orphan designation: for: Treatment of pseudomyxoma peritonei (updated)
Date of first decision: 14/12/2018, Positive, Last updated: 05/02/2020 -
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Orphan designation: for: Treatment of neurodegeneration with brain iron accumulation (updated)
Date of first decision: 27/06/2018, Positive, Last updated: 04/02/2020