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Orphan designation: Cannabidiol, Treatment of Dravet syndrome
Case Medical Research
◽
10.31525/cmr-10e0aa0
◽
2019
◽
Author(s):
Keyword(s):
Dravet Syndrome
◽
Orphan Designation
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References
Orphan designation: Cannabidiol, Treatment of Dravet syndrome
Case Medical Research
◽
10.31525/cmr-10e76a4
◽
2019
◽
Author(s):
Keyword(s):
Dravet Syndrome
◽
Orphan Designation
Download Full-text
Orphan designation: Fenfluramine hydrochloride, Treatment of Dravet syndrome
Case Medical Research
◽
10.31525/cmr-14481fb
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2019
◽
Author(s):
Keyword(s):
Dravet Syndrome
◽
Orphan Designation
Download Full-text
Mutation analysis of the SCN1A gene for suspected Dravet syndrome/GEFS+ – High detection yield, unsatisfactory prognostic value
Neuropediatrics
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10.1055/s-0029-1215751
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2008
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Vol 39
(05)
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Author(s):
N Kohlschmidt
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CD Gross
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S Kuczaty
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R Sassen
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W Kunz
Keyword(s):
Mutation Analysis
◽
Prognostic Value
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Dravet Syndrome
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High Detection
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Scn1a Gene
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Possible dysregulation of the endocannabinoid system in a cohort of patients with Dravet syndrome
10.26226/morressier.5785edc6d462b80296c99338
◽
2016
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Author(s):
Valentina Satta
Keyword(s):
Endocannabinoid System
◽
Dravet Syndrome
Download Full-text
Orphan designation: Anti-neonatal Fc receptor human monoclonal antibody, Prevention of haemolytic disease of the foetus and newborn
Case Medical Research
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10.31525/cmr-277ad45
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2020
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Author(s):
Keyword(s):
Monoclonal Antibody
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Human Monoclonal Antibody
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Fc Receptor
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Neonatal Fc Receptor
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Haemolytic Disease
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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
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10.31525/cmr-277ad3d
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2020
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Author(s):
Keyword(s):
Primary Biliary Cirrhosis
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Biliary Cirrhosis
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Orphan Designation
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Chloride Treatment
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Methyl Phenyl
Download Full-text
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
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10.31525/cmr-27659a5
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2020
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Author(s):
Keyword(s):
Carboxylic Acid
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Soft Tissue
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Soft Tissue Sarcoma
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Orphan Designation
Download Full-text
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
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10.31525/cmr-276599f
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2020
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Author(s):
Keyword(s):
Graft Versus Host Disease
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Sulfonic Acid
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Acid Salt
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Salt Treatment
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Methane Sulfonic Acid
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Host Disease
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Graft Versus Host
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Orphan Designation
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Orphan designation: 18-(p-(131I)-iodophenyl)octadecyl phosphocholine, Treatment of multiple myeloma
Case Medical Research
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10.31525/cmr-276599d
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2020
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Author(s):
Keyword(s):
Multiple Myeloma
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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
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10.31525/cmr-134bcab
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2019
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Author(s):
Keyword(s):
Gastrointestinal Stromal Tumours
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Orphan Designation
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