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Orphan designation: Adeno-associated virus serotype R100 containing the human RPGRorf15 gene isoform for: Treatment of inherited retinal dystrophies due to defects in the RPGR gene
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Cannabidiol for: Treatment of epidermolysis bullosa
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD123 for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment for: Treatment of focal segmental glomerulosclerosis
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Elezanumab for: Treatment of spinal cord injury
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Streptococcus pyogenes, group A, type 3, strain Su, inactivated for: Treatment of lymphatic malformations
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Elamipretide for: Treatment of myopathic mitochondrial DNA depletion syndrome
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Ibudilast for: Treatment of fragile X syndrome
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Adeno-associated virus serotype 8 expressing the human gamma-sarcoglycan gene for: Treatment of limb-girdle muscular dystrophy
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Autologous peripheral blood-derived CD4 T-cells CRISPR-edited at the CD40LG locus for: Treatment of hyper IgM syndromes
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Icerguastat acetate for: Treatment of alopecia totalis
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: Chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: pasireotide for: Treatment of noninsulinoma pancreatogenous hypoglycemia syndrome
Date of designation: 16/05/2022, Positive, Last updated: 14/12/2022