89 results
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Orphan designation: for: Treatment of peripheral T-cell lymphoma (updated)
Date of first decision: 21/09/2019, Positive, Last updated: 16/01/2020 -
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Orphan designation: for: Treatment of ovarian cancer
Date of first decision: 04/08/2010, Positive, Last updated: 18/12/2019 -
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Orphan designation: for: Treatment of hypophosphatasia
Date of first decision: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: for: Treatment of lysosomal acid lipase deficiency
Date of first decision: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: for: Treatment of acute myeloid leukaemia
Date of first decision: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: for: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: for: Treatment of argininosuccinic aciduria
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: for: Treatment of citrullinaemia type 1
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: for: Treatment of ornithine carbamoyltransferase deficiency
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: for: Treatment of multiple myeloma
Date of first decision: 08/10/2009, Positive, Last updated: 18/06/2019 -
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Orphan designation: for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: for: Treatment of hyperphenylalaninaemia
Date of first decision: 28/01/2010, Positive, Last updated: 29/05/2019 -
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Orphan designation: for: Treatment of mucormycosis
Date of first decision: 04/06/2014, Positive, Last updated: 21/05/2019 -
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Orphan designation: for: Treatment of invasive aspergillosis
Date of first decision: 04/07/2014, Positive, Last updated: 21/05/2019 -
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Orphan designation: for: Treatment of Fabry disease
Date of first decision: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: for: Treatment of familial chylomicronaemia syndrome
Date of first decision: 19/02/2014, Positive, Last updated: 20/05/2019 -
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Orphan designation: for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: for: Treatment of retinitis pigmentosa
Date of first decision: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: for: Treatment of Leber's congenital amaurosis
Date of first decision: 02/04/2012, Positive, Last updated: 04/03/2019 -
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Orphan designation: for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 14/10/2016, Positive, Last updated: 28/01/2019 -
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Orphan designation: for: Treatment of neuroblastoma
Date of first decision: 08/11/2012, Positive, Last updated: 22/01/2019 -
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Orphan designation: for: Treatment of cystic fibrosis
Date of first decision: 27/02/2017, Positive, Last updated: 22/11/2018 -
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Orphan designation: for: Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)
Date of first decision: 24/07/2009, Positive, Last updated: 24/10/2018