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31 results
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of first decision: 18/12/2002, Positive, Last updated: 12/04/2016 -
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Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of first decision: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: idebenone for: Treatment of Duchenne muscular dystrophy
Date of first decision: 20/03/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of first decision: 08/05/2008, Positive, Last updated: 11/04/2016 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase for: Treatment of lipoprotein-lipase deficiency
Date of first decision: 09/03/2004, Positive, Last updated: 17/10/2013