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Orphan designation: Human glucagon-like peptide-2 analogue linked to a human immunoglobulin Fc fragment for: Treatment of short bowel syndrome
Date of designation: 11/01/2019, Positive, Last updated: 27/03/2019 -
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Orphan designation: Benserazide hydrochloride for: The treatment of sickle cell disease
Date of designation: 11/01/2019, Positive, Last updated: 27/03/2019 -
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Orphan designation: Acetylleucine for: Treatment of ataxia telangiectasia
Date of designation: 11/01/2019, Positive, Last updated: 25/04/2019 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for: Treatment of beta thalassaemia intermedia and major
Date of designation: 11/01/2019, Positive, Last updated: 27/03/2019 -
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Orphan designation: melatonin for: Treatment of perinatal asphyxia
Date of designation: 11/01/2019, Positive, Last updated: 28/06/2019 -
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Orphan designation: Vinorelbine tartrate for: Treatment of soft tissue sarcoma
Date of designation: 11/01/2019, Positive, Last updated: 27/03/2019 -
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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: Mercaptamine-pantetheine disulfide for: Treatment of Rett syndrome
Date of designation: 11/01/2019, Positive, Last updated: 27/03/2019 -
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Orphan designation: Rozanolixizumab for: Treatment of immune thrombocytopenia
Date of designation: 11/01/2019, Withdrawn, Last updated: 08/05/2023 -
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Orphan designation: miglustat for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 11/01/2019, Withdrawn, Last updated: 24/03/2023