Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran), Treatment of acute hepatic porphyria

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: 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: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran), Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-cc9827 ◽

2019 ◽

Author(s):

Keyword(s):

Primary Hyperoxaluria ◽

Orphan Designation ◽

Covalently Linked ◽

Sirna Oligonucleotide

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran), Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-cc49d5 ◽

2019 ◽

Author(s):

Keyword(s):

Primary Hyperoxaluria ◽

Orphan Designation ◽

Covalently Linked ◽

Sirna Oligonucleotide

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Orphan designation: synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-de550b ◽

2019 ◽

Author(s):

Keyword(s):

Lactate Dehydrogenase ◽

Primary Hyperoxaluria ◽

Modified Nucleosides ◽

Orphan Designation ◽

Sirna Oligonucleotide

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Orphan designation: synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of primary hyperoxaluria

Case Medical Research ◽

10.31525/cmr-de7557 ◽

2019 ◽

Author(s):

Keyword(s):

Lactate Dehydrogenase ◽

Primary Hyperoxaluria ◽

Modified Nucleosides ◽

Orphan Designation ◽

Sirna Oligonucleotide

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Orphan designation: Poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain, Treatment of paroxysmal nocturnal haemoglobinuria

Case Medical Research ◽

10.31525/cmr-110cf65 ◽

2019 ◽

Author(s):

Keyword(s):

Polyethylene Glycol ◽

Synthetic Peptide ◽

Paroxysmal Nocturnal Haemoglobinuria ◽

Orphan Designation ◽

Covalently Linked

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues, Treatment of congenital alpha-1 antitrypsin deficiency

Case Medical Research ◽

10.31525/cmr-2a47e1d ◽

2020 ◽

Author(s):

Keyword(s):

Modified Nucleosides ◽

Orphan Designation ◽

Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin Deficiency ◽

Alpha 1 Antitrypsin ◽

Sirna Oligonucleotide

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Orphan designation: Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain, Treatment of C3 glomerulopathy

Case Medical Research ◽

10.31525/cmr-2704234 ◽

2020 ◽

Author(s):

Keyword(s):

Polyethylene Glycol ◽

Synthetic Peptide ◽

C3 Glomerulopathy ◽

Orphan Designation ◽

Covalently Linked

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran), Prevention of delayed graft function after renal transplantation

Case Medical Research ◽

10.31525/cmr-1a95682 ◽

2019 ◽

Author(s):

Keyword(s):

Renal Transplantation ◽

Graft Function ◽

Delayed Graft Function ◽

Orphan Designation ◽

Sirna Oligonucleotide ◽

P53 Mrna

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