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22 results
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
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Orphan designation: Escherichia coli, strain Nissle 1917, expressing high affinity phenylalanine transporter, phenylalanine ammonia lyase and L-amino acid deaminase for: Treatment of hyperphenylalaninaemia
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: losartan for: Treatment of osteogenesis imperfecta
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: Doxorubicin for: Treatment of soft tissue sarcoma
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: Sebetralstat for: Treatment of hereditary angioedema
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: amitriptyline for: Treatment of erythromelalgia
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: ropeginterferon alfa-2b for: Treatment of chronic myeloid leukaemia
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid for: Treatment of gain-of-function mutations of STIM1 and ORAI1 related diseases
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: 4-[[(3S)-1-benzylpyrrolidin-3-yl]-methylamino]-2-fluoro-5-methyl-N-(1,3-thiazol-4-yl)benzenesulfonamide for: Treatment of SCN8A developmental and epileptic encephalopathy
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 24/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: Sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z)10,17-dihydroxy-docosa-4,7,11,13,15,19-hexaenoate for: Prevention of retinopathy of prematurity
Date of designation: 21/06/2022, Positive, Last updated: 15/09/2022 -
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Orphan designation: Lithium carbonate for: treatment of TBR1-related disorder
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Sirolimus for: Treatment of lymphatic malformations
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA for: Treatment of Angelman syndrome
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Human allogeneic keratinocytes for: Treatment of partial deep dermal and full thickness burns
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671 for: Treatment of methylmalonic acidaemia
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: 2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl -(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl for: Treatment of cystic fibrosis
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Eltanexor for: Treatment of myelodysplastic syndromes
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Humanised IgG4 monoclonal antibody against A proliferation-inducing ligand for: Treatment of primary IgA nephropathy
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Sirolimus for: Treatment of follicular lymphoma
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Diflunisal for: Treatment of ATTR amyloidosis
Date of designation: 21/06/2022, Positive, Last updated: 31/10/2022 -
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Orphan designation: Govorestat for: Treatment of galactosaemia (updated)
Date of designation: 21/06/2022, Positive, Last updated: 29/03/2023