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11 results
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Orphan designation: Octreotide acetate for: Treatment of carcinoid syndrome
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Vorasidenib hemicitrate hemihydrate for: Treatment of glioma
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid for: Treatment of Duchenne muscular dystrophy
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: N1,N14-diethyl-3S,12S-dihydroxyhomospermine tetrahydrochloride for: Treatment of pancreatic cancer
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Autologous hematopoietic cells genetically modified with a lentiviral vector containing the human RAG2 gene for: Treatment of recombination-activating gene 2 deficient severe combined immunodeficiency
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Nanatinostat, Valganciclovir for: Treatment of diffuse large B-cell lymphoma
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Retifanlimab for: Treatment of Merkel cell carcinoma
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Efzofitimod for: Treatment of sarcoidosis
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Opelconazole for: Treatment of invasive aspergillosis
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Adeno-associated viral vector serotype 2 containing the human SLC6A3 gene for: Treatment of dopamine transporter deficiency syndrome
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene for: Treatment of chronic granulomatous disease
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023