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14 results
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Orphan designation: Delpazolid for: Treatment of tuberculosis
Date of designation: 10/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Copper nanocluster conjugated to acetate, histidinate and ascorbate for: Treatment of Menkes disease
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of AL amyloidosis
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: N-(4-(4-amino-5-(3-fluoro-4-((4-methylpyrimidin-2-yl)oxy)phenyl)-7-methyl-7H-pyrrolo[2,3-d] pyrimidin-6-yl)phenyl)methacrylamide hydrochloride for: Treatment of biliary tract cancer
Date of designation: 10/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Adeno-associated virus serotype 5 vector encoding C1-esterase inhibitor for: Treatment of hereditary angioedema
Date of designation: 10/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Propranolol for: Treatment of familial cerebral cavernous malformations
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: atorvastatin for: Treatment of familial cerebral cavernous malformations
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: rovatirelin for: Treatment of spinocerebellar ataxia
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Pegcetacoplan for: Treatment of C3 glomerulopathy with or without immune complexes
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Refusal of orphan designation for the treatment of retinopathy of prematurity
Date of refusal of designation: 28/11/2022, Negative, Last updated: 23/01/2023 -
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Orphan designation: tricaprilin for: Treatment of West syndrome
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Cannabidiol for: Treatment of 22q11.2 deletion syndrome
Date of designation: 11/11/2022, Positive, Last updated: 07/03/2023 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human ABCD1 gene for: Treatment of adrenoleukodystrophy
Date of designation: 11/11/2022, Positive, Last updated: 25/01/2023 -
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Orphan designation: (R)-deuteropioglitazone hydrochloride for: Treatment of adrenoleukodystrophy
Date of designation: 11/11/2022, Positive, Last updated: 23/01/2023