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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
Download Full-text
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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
Case Medical Research
◽
10.31525/cmr-1e36742
◽
2019
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Author(s):
Keyword(s):
Orphan Designation
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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
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10.31525/cmr-ea7c31
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2019
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Author(s):
Keyword(s):
Orphan Designation
◽
Beta Thalassaemia
◽
Covalently Linked
◽
Thalassaemia Intermedia
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Sirna Oligonucleotide
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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
Download Full-text
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
Download Full-text
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
Download Full-text
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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