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14 results
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Orphan designation: Upifitamab rilsodotin for: Treatment of ovarian cancer
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: 16-base single stranded RNA targeting miR-23b linked to oleic acid for: Treatment of myotonic disorders
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of ATTR amyloidosis
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Mebendazole for: Treatment of autosomal dominant polycystic kidney disease
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Copper (64Cu) oxodotreotide for: Diagnosis of neuroendocrine neoplasms
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Utreloxastat for: Treatment of amyotrophic lateral sclerosis
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Recombinant adeno-associated virus Olig001 containing human aspartoacylase cDNA for: Treatment of Canavan disease
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Duvelisib for: Treatment of peripheral T-cell lymphoma
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Serplulimab for: Treatment of small cell lung cancer
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Autologous T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against CD7 for: Treatment of acute lymphoblastic leukaemia
Date of designation: 09/12/2022, Positive, Last updated: 14/03/2023 -
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Orphan designation: Briquilimab for: Treatment in haematopoietic stem cell transplantation
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo-5-phenyl-6,7-dihydrothieno[2,3-b]pyridin-4-olate monohydrate for: Treatment of adrenoleukodystrophy
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: (2S)-4-[2-methoxyethyl-[4-(5,6,7,8-tetrahydro-1,8-naphthyridin-2-yl)butyl]amino]-2-(quinazolin-4-ylamino)butanoic acid for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023 -
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Orphan designation: Adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 09/12/2022, Positive, Last updated: 13/03/2023