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11 results
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Orphan designation: Lurbinectedin for: Treatment of small cell lung cancer
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Autologous adult live cultured osteoblasts for: Treatment of non-traumatic osteonecrosis
Date of designation: 26/02/2019, Positive, Last updated: 27/08/2020 -
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Orphan designation: Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Positive, Last updated: 04/05/2021 -
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Orphan designation: 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester for: Treatment of infantile neuroaxonal dystrophy
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Lentiviral vector encoding human coagulation factor IX for: Treatment of haemophilia B
Date of designation: 26/02/2019, Positive, Last updated: 02/12/2022 -
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Orphan designation: losartan for: Treatment of epidermolysis bullosa
Date of designation: 26/02/2019, Positive, Last updated: 07/03/2022 -
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Orphan designation: Poly(N-acetyl, N-arginyl)glucosamine for: Treatment of cystic fibrosis
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Allogeneic cultured postnatal thymus-derived tissue for: Treatment of DiGeorge syndrome
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Allogeneic cultured postnatal thymus-derived tissue for: Treatment of CHARGE syndrome
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019 -
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Orphan designation: Allogeneic cultured postnatal thymus-derived tissue for: Treatment of severe combined immunodeficiency due to FOXN1 deficiency
Date of designation: 26/02/2019, Positive, Last updated: 09/04/2019