Effects of cyclodextrin in two patients with Niemann-Pick Type C disease

Molecular Genetics and Metabolism

Volumn 108, Issue 1, 2013, Pages 76-81

  Matsuo, Muneaki ^a   Togawa, Masami ^b   Hirabaru, Keiko ^a   Mochinaga, Sakiko ^c   Narita, Aya ^b   Adachi, Masao ^d   Egashira, Masakazu ^a   Irie, Tetsumi ^e   Ohno, Kousaku ^b  

^a SAGA UNIVERSITY   (Japan)

^b TOTTORI UNIVERSITY   (Japan)

^c SAGA UNIVERSITY HOSPITAL   (Japan)

^d Kakogawa West City Hospital   (Japan)

^e KUMAMOTO UNIVERSITY   (Japan)

Author keywords

Cholesterol; Cyclodextrin; Niemann Pick Type C disease

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN;

ADOLESCENT; ARTICLE; ATAXIA; CASE REPORT; CATAPLEXY; CHILD; CLINICAL EFFECTIVENESS; COMPUTER ASSISTED TOMOGRAPHY; CONVULSION; DISEASE SEVERITY; DRUG DOSE INCREASE; DRUG EFFICACY; DYSARTHRIA; DYSPHAGIA; ELECTROENCEPHALOGRAPHY; EVOKED BRAIN STEM AUDITORY RESPONSE; FEMALE; GAZE PARALYSIS; GENE MUTATION; HEPATOSPLENOMEGALY; HUMAN; LUMBAR SCOLIOSIS; MODIFIED DISABILITY SCALE; NIEMANN PICK DISEASE; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PRESCHOOL CHILD; PRIORITY JOURNAL; RATING SCALE; SCOLIOSIS; TRACHEOSTOMY; URINALYSIS;

ADOLESCENT; BETA-CYCLODEXTRINS; CHILD, PRESCHOOL; FEMALE; HUMANS; NIEMANN-PICK DISEASE, TYPE C;

MUS;

EID: 84871717628     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2012.11.005     Document Type: Article

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