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Orphan designation: (S)-12-fluoro-4-(2-methylpyridin-3-yl)-7a,8,13,14-tetrahydro-7H-[1,2,4]triazolo[4',3':1,6]pyrido[3,2-b]benzofuro[4,3-fg][1,4]oxazonine for: Treatment of sickle cell disease
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Emactuzumab for: Treatment of tenosynovial giant cell tumour, localised and diffuse type
Date of designation: 16/03/2022, Positive, Last updated: 20/06/2022 -
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Orphan designation: Heterologous human adult liver-derived stem cells for: Treatment of argininosuccinic aciduria
Date of designation: 16/03/2022, Positive, Last updated: 20/06/2022 -
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Orphan designation: Dersimelagon for: Treatment of erythropoietic protoporphyria
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Codergocrine mesilate, oxitriptan for: Treatment of fragile X syndrome
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Treprostinil sodium for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Hymecromone for: Treatment of spinal cord injury
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Tropatepine hydrochloride for: Treatment of narcolepsy
Date of designation: 16/03/2022, Positive, Last updated: 16/06/2022 -
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Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase for: Treatment of phenylalanine hydroxylase deficiency
Date of designation: 16/03/2022, Positive, Last updated: 11/10/2022 -
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Orphan designation: Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase for: Treatment of mucopolysaccharidosis type II (Hunter Syndrome)
Date of designation: 16/03/2022, Positive, Last updated: 11/10/2022 -
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Orphan designation: Alisporivir for: Treatment of collagen VI-related myopathies
Date of designation: 16/03/2022, Positive, Last updated: 14/12/2022