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19 results
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Orphan designation: Oxygen, sodium chloride solution 0.9% for: Treatment of amyotrophic lateral sclerosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Crofelemer for: Treatment of microvillus inclusion disease
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Autologous T cells transduced with a lentiviral vector expressing a chimeric antigen receptor against CLL-1 for: Treatment of acute myeloid leukaemia
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Epeleuton for: Treatment of sickle cell disease
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Velusetrag for: Treatment of intestinal pseudo-obstruction
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Inaxaplin for: Treatment of apolipoprotein L1-mediated kidney disease
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Adeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence for: Treatment of otoferlin gene-mediated hearing loss
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Aglatimagene besadenovec for: Treatment of glioma
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Adeno-associated viral vector serotype rh.10 encoding the CLN2 gene for: Treatment of neuronal ceroid lipofuscinosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: isotretinoin for: Treatment of autosomal recessive congenital ichthyosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Human IgG4k monoclonal antibody against CD89 for: Treatment of linear IgA bullous dermatosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Etidronate disodium for: Treatment of pseudoxanthoma elasticum
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Sodium ({(2S)-1,4-bis[2-(4-chloro-3-fluorophenoxy)acetamido]bicyclo[2.2.2]octan-2-yl}oxy)methyl hydrogen phosphate-2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1/1) for: Treatment of vanishing white matter disease
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Selinexor for: Treatment of myelofibrosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Methyl(R)-4-((3S,5R,7R,8R,9S,10S,13R,14S,17R)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-3-ylmethyl)amino)butyl)amino)hexadecahydro-1H-cyclopenta[a]phenanthren-17-yl)pentanoate for: Treatment of Rett syndrome
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Eculizumab for: Treatment of Guillain-Barré syndrome
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Acetylleucine for: Treatment of GM1 gangliosidosis
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: ropeginterferon alfa-2b for: Treatment of essential thrombocythaemia
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Refusal of orphan designation for the prevention of spaceflight-related radiation and microgravity
Date of refusal of designation: 14/10/2022, Negative, Last updated: 23/01/2023