Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen), Treatment of ATTR amyloidosis

Case Medical Research ◽

10.31525/cmr-12e1a9e ◽

2019 ◽

Author(s):

Keyword(s):

Phosphorothioate Oligonucleotide ◽

Orphan Designation ◽

Attr Amyloidosis

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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen), Treatment of familial chylomicronaemia syndrome

Case Medical Research ◽

10.31525/cmr-132156a ◽

2019 ◽

Author(s):

Keyword(s):

Phosphorothioate Oligonucleotide ◽

Orphan Designation ◽

Apolipoprotein C

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Orphan designation: 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid, Treatment of ATTR amyloidosis

Case Medical Research ◽

10.31525/cmr-babd63 ◽

2019 ◽

Author(s):

Keyword(s):

Acid Treatment ◽

Orphan Designation ◽

Attr Amyloidosis

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Orphan designation: 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid, Treatment of ATTR amyloidosis

Case Medical Research ◽

10.31525/cmr-bb17b2 ◽

2019 ◽

Author(s):

Keyword(s):

Acid Treatment ◽

Orphan Designation ◽

Attr Amyloidosis

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Orphan designation: Anti-neonatal Fc receptor human monoclonal antibody, Prevention of haemolytic disease of the foetus and newborn

Case Medical Research ◽

10.31525/cmr-277ad45 ◽

2020 ◽

Author(s):

Keyword(s):

Monoclonal Antibody ◽

Human Monoclonal Antibody ◽

Fc Receptor ◽

Neonatal Fc Receptor ◽

Haemolytic Disease ◽

Orphan Designation

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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride, Treatment of primary biliary cirrhosis

Case Medical Research ◽

10.31525/cmr-277ad3d ◽

2020 ◽

Author(s):

Keyword(s):

Primary Biliary Cirrhosis ◽

Biliary Cirrhosis ◽

Orphan Designation ◽

Chloride Treatment ◽

Methyl Phenyl

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Orphan designation: 4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide, Treatment of soft tissue sarcoma

Case Medical Research ◽

10.31525/cmr-27659a5 ◽

2020 ◽

Author(s):

Keyword(s):

Carboxylic Acid ◽

Soft Tissue ◽

Soft Tissue Sarcoma ◽

Orphan Designation

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Orphan designation: 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt, Treatment of graft-versus-host disease

Case Medical Research ◽

10.31525/cmr-276599f ◽

2020 ◽

Author(s):

Keyword(s):

Graft Versus Host Disease ◽

Sulfonic Acid ◽

Acid Salt ◽

Salt Treatment ◽

Methane Sulfonic Acid ◽

Host Disease ◽

Graft Versus Host ◽

Orphan Designation

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Orphan designation: 18-(p-(131I)-iodophenyl)octadecyl phosphocholine, Treatment of multiple myeloma

Case Medical Research ◽

10.31525/cmr-276599d ◽

2020 ◽

Author(s):

Keyword(s):

Multiple Myeloma ◽

Orphan Designation

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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine (avapritinib) , Treatment of gastrointestinal stromal tumours

Case Medical Research ◽

10.31525/cmr-134bcab ◽

2019 ◽

Author(s):

Keyword(s):

Gastrointestinal Stromal Tumours ◽

Orphan Designation

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Orphan designation: Fibrinogen-coated albumin spheres, Treatment of Ebola virus disease

Case Medical Research ◽

10.31525/cmr-134bca7 ◽

2019 ◽

Author(s):

Keyword(s):

Ebola Virus ◽

Virus Disease ◽

Ebola Virus Disease ◽

Orphan Designation

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