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Orphan designation: Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor for: Treatment of Duchenne muscular dystrophy
Date of designation: 19/11/2019, Positive, Last updated: 28/04/2023 -
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Orphan designation: 4-((E)-(5-(2-(2-((S)-2-((S)-1-(L-threonyl-L-lysyl)pyrrolidine-2-carboxamido)-5-guanidinopentanamido)acetamido)-2-carboxyethyl)-2-hydroxyphenyl)diazenyl)phenyl (2-(trimethylammonio)ethyl) phosphate for: Treatment of non-infectious uveitis
Date of designation: 13/11/2019, Positive, Last updated: 12/02/2020 -
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Orphan designation: (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol for: Treatment of GM2 gangliosidosis
Date of designation: 13/11/2019, Positive, Last updated: 12/02/2020 -
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Orphan designation: Camsirubicin for: Treatment of soft tissue sarcoma
Date of designation: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: Chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4 for: Treatment of AL amyloidosis
Date of designation: 13/11/2019, Positive, Last updated: 28/01/2022 -
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Orphan designation: Exendin (9-39) for: Treatment of congenital hyperinsulinism
Date of designation: 13/11/2019, Positive, Last updated: 13/02/2020 -
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Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme (Inetagugene geperpavec) for: Treatment of autosomal recessive congenital ichthyosis
Date of designation: 13/11/2019, Positive, Last updated: 13/01/2023 -
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Orphan designation: Ganaxolone for: Treatment of CDKL5 deficiency disorder
Date of designation: 13/11/2019, Positive, Last updated: 31/07/2023 -
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Orphan designation: autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel) for: Treatment of mantle cell lymphoma
Date of designation: 13/11/2019, Positive, Last updated: 15/09/2021