Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease

Molecular Genetics and Metabolism Reports

Volumn 1, Issue , 2014, Pages 391-400

  Matsuo, Muneaki ^a   Shraishi, Koki ^b   Wada, Koki ^c   Ishitsuka, Yoichi ^b   Doi, Hirohito ^a   Maeda, Miyuki ^a   Mizoguchi, Tatsuhiro ^a   Eto, Junya ^a   Mochinaga, Sakiko ^a   Arima, Hidetoshi ^b   Irie, Tetsumi ^b  

^a SAGA UNIVERSITY   (Japan)

^b KUMAMOTO UNIVERSITY   (Japan)

^c Fuji   (Japan)

Author keywords

Cholesterol; Cyclodextrin; Intracerebroventricular administration; Intrathecal administration; Niemann Pick type C disease; Pharmacokinetics

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; CHOLESTEROL; CHOLINE; CREATINE; MIGLUSTAT; N ACETYLASPARTIC ACID; TAU PROTEIN;

ABSENCE OF SIDE EFFECTS; ADD ON THERAPY; ARTICLE; ATAXIA; BRAIN ATROPHY; CASE REPORT; CATAPLEXY; CEREBROSPINAL FLUID ANALYSIS; CHILD; CHOLESTEROL BLOOD LEVEL; CONVULSION; DYSARTHRIA; DYSPHAGIA; FEMALE; GASTROSTOMY; HEPATOSPLENOMEGALY; HUMAN; IN VITRO STUDY; NIEMANN PICK DISEASE; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PERINATAL PERIOD; POSITRON EMISSION TOMOGRAPHY;

EID: 84916599122     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2014.08.004     Document Type: Article

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