Orphan designation: 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridine-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride, Treatment of beta-thalassaemia intermedia and major

Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-ea7c31 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Covalently Linked ◽

Thalassaemia Intermedia ◽

Sirna Oligonucleotide

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Orphan designation: human apotransferrin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-bb17bc ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-ea2d19 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Covalently Linked ◽

Thalassaemia Intermedia ◽

Sirna Oligonucleotide

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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene, Treatment of beta thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-de550e ◽

2019 ◽

Author(s):

Keyword(s):

Stem Cells ◽

Lentiviral Vector ◽

Globin Gene ◽

Haematopoietic Stem Cells ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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Orphan designation: human apotransferrin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-bc10f9 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

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Orphan designation: Bitopertin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-3996c1 ◽

2018 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

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Orphan designation: Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene, Treatment of beta thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-12043ed ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Globin Gene ◽

Beta Globin ◽

Orphan Designation ◽

Beta Globin Gene ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Cd34 Cells ◽

Thalassaemia Intermedia

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Orphan designation: 1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-6a72a7 ◽

2018 ◽

Author(s):

Keyword(s):

Salt Treatment ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Acetate Salt ◽

Thalassaemia Intermedia

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Orphan designation: Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-277ad49 ◽

2020 ◽

Author(s):

Keyword(s):

Stem Cells ◽

Haematopoietic Stem Cells ◽

Enhancer Region ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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Orphan designation: 1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-6ac705 ◽

2018 ◽

Author(s):

Keyword(s):

Salt Treatment ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Acetate Salt ◽

Thalassaemia Intermedia

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