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17 results
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Orphan designation: Glofitamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Human IgG1 monoclonal antibody against sortilin for: Treatment of frontotemporal dementia
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Devimistat for: Treatment of Burkitt's lymphoma
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Batiraxcept for: Treatment of ovarian cancer
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Humanised IgG1 monoclonal antibody against SEZ6 linked to N-acetyl-calicheamicin for: Treatment of small cell lung cancer
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Ganaxolone for: Treatment of tuberous sclerosis
Date of designation: 15/10/2021, Positive, Last updated: 20/01/2022 -
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Orphan designation: Mocravimod for: Treatment in haematopoietic stem cell transplantation
Date of designation: 15/10/2021, Positive, Last updated: 13/04/2022 -
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Orphan designation: Adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene for: Treatment of cone-rod dystrophy
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Allogeneic umbilical cord mesenchymal cells-derived extracellular vesicles for: Prevention of bronchopulmonary dysplasia
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Selinexor for: Treatment of glioma
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Psilocybine for: Treatment of fragile X syndrome
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Encaleret sulfate for: Treatment of hypoparathyroidism
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Idursulfase beta for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 15/10/2021, Positive, Last updated: 21/01/2022 -
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Orphan designation: Autologous haematopoietic stem and progenitor cell population containing CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA ex vivo expanded for: Treatment of osteopetrosis
Date of designation: 15/10/2021, Positive, Last updated: 07/11/2022 -
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Orphan designation: Adeno-associated virus of serotype rh10 encoding Human MLC1 under the control of GFAP promoter for: Treatment of megalencephalic leukoencephalopathy with subcortical cysts
Date of designation: 15/10/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Adeno-associated virus serotype rh10 containing the human GALC gene for: Treatment of Krabbe disease
Date of designation: 15/10/2021, Positive, Last updated: 18/11/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I
Date of designation: 15/10/2021, Withdrawn, Last updated: 20/12/2022