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13 results
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Orphan designation: Norucholic acid for: Treatment of primary biliary cholangitis
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Adeno-associated virus vector serotype 9 encoding human gigaxonin gene for: Treatment of giant axonal neuropathy
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: N-(methoxypolyethylene glycol 5000 carbamoyl)-1,2-dipalmitoyl-sn-glycero-3-phosphatidylethanolamine for: Treatment in solid organ transplantation
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Devimistat for: Treatment of biliary tract cancer
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Obecabtagene autoleucel for: Treatment of acute lymphoblastic leukaemia
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter for: Treatment of haemophilia A
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Adeno-associated virus serotype C102 containing the human GLA gene for: Treatment of Fabry disease
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: tiratricol for: Treatment of resistance to thyroid hormone type beta
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: for: Treatment of primary sclerosing cholangitis
Date of designation: 13/04/2022, Positive, Last updated: 19/07/2022 -
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Orphan designation: Cannabidiol for: Treatment of epilepsy with myoclonic-atonic seizures
Date of designation: 13/04/2022, Positive, Last updated: 16/06/2023 -
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Orphan designation: Glofitamab for: Treatment of mantle cell lymphoma
Date of designation: 13/04/2022, Positive, Last updated: 18/07/2022 -
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Orphan designation: Adeno-associated virus serotype 9 containing human MYBPC3 gene for: Treatment of Hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene encoding cardiac myosin-binding protein C
Date of designation: 13/04/2022, Positive, Last updated: 18/07/2022 -
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Orphan designation: Belzupacap sarotalocan for: Treatment of uveal melanoma
Date of designation: 13/04/2022, Last updated: 22/09/2023