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22 results
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Orphan designation: Efgartigimod alfa for: Treatment of myasthenia gravis
Date of designation: 10/08/2022, Positive, Last updated: 12/09/2022 -
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Orphan designation: Autologous CD34+ haematopoietic stem and progenitor cells genetically modified with a lentiviral vector encoding for the N-acetylgalactosamine 6-sulfatase cDNA for: Treatment of mucopolysaccharidosis type IV A (Morquio A syndrome)
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Toll-like receptor 4 agonist for: Treatment of osteosarcoma
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Nanatinostat, Valganciclovir for: Treatment of peripheral T-cell lymphoma
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Lithium carbonate for: Treatment of familial adenomatous polyposis (FAP)
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Bezafibrate for: Treatment of primary sclerosing cholangitis
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Mazindol for: Treatment of idiopathic hypersomnia
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for: Treatment of haemophilia A
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Calmangafodipir for: Prevention of acute liver failure
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Allogeneic placenta-derived decidual stromal cells for: Treatment of graft-versus-host-disease
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Epetraborole for: Treatment of nontuberculous mycobacterial lung disease
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Autologous human bone marrow-derived haematopoietic and mesenchymal stem cells depleted of erythrocytes, monocytes and lymphocytes for: Treatment of frontotemporal dementia
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Vutrisiran for: Treatment of Stargardt's disease
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Sotuletinib for: Treatment of amyotrophic lateral sclerosis
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Batoclimab for: Treatment of myasthenia gravis
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Allogeneic adult liver-derived stem cells for: Treatment of urea cycle disorders
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Gold for: Treatment of amyotrophic lateral sclerosis
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: clarithromycin, clofazimine, rifabutin for: Treatment of nontuberculous mycobacterial lung disease
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Tamibarotene for: Treatment of myelodysplastic syndromes
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: (7S)-8,8-dimethyl-7-{[(2E)-3-phenyl-2-propen-1-yl]oxy}-7,8-dihydro-2H,6H-pyrano[3,2-g]chromen-2-one for: Treatment of Hutchinson-Gilford progeria syndrome
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Humanised IgG1 tetravalent monoclonal antibody against death receptor 5 for: Treatment of chondrosarcoma
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022 -
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Orphan designation: Amphotericin B for: Treatment of cryptococcosis
Date of designation: 10/08/2022, Positive, Last updated: 15/12/2022