Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1

Molecular Genetics and Metabolism

Volumn 116, Issue 1-2, 2015, Pages 75-79

  Maarup, Timothy J ^a   Chen, Agnes H ^a   Porter, Forbes D ^b   Farhat, Nicole Y ^b   Ory, Daniel S ^c   Sidhu, Rohini ^c   Jiang, Xuntian ^c   Dickson, Patricia I ^a  

^a HARBOR UCLA MEDICAL CENTER   (United States)

^b EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^c WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Cyclodextrin; Niemann Pick C; Ototoxicity; Therapeutic

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; 24 HYDROXYCHOLESTEROL; BIOLOGICAL MARKER; OXYSTEROL; UNCLASSIFIED DRUG; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; 24-HYDROXYCHOLESTEROL; BETA CYCLODEXTRIN DERIVATIVE; CHOLESTEROL DERIVATIVE; EXCIPIENT;

ARTICLE; CASE REPORT; CENTRAL NERVOUS SYSTEM; CEREBROSPINAL FLUID; CHILD; CLINICAL EVALUATION; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; HEARING IMPAIRMENT; HEPATOSPLENOMEGALY; HUMAN; INJECTION SITE PAIN; JAUNDICE; LUMBAR PUNCTURE; MALE; NEUROIMAGING; NIEMANN PICK C1 DISEASE; NIEMANN PICK DISEASE; OXIDATIVE STRESS; PRIORITY JOURNAL; SCHOOL CHILD; SYMPTOM; TREATMENT RESPONSE; BLOOD; DISEASE COURSE; DRUG EFFECTS; EYE MOVEMENT; HEARING; INTRASPINAL DRUG ADMINISTRATION; METABOLISM; NIEMANN-PICK DISEASE, TYPE C; PATHOPHYSIOLOGY;

BETA-CYCLODEXTRINS; BIOMARKERS; CHILD; DISEASE PROGRESSION; EXCIPIENTS; EYE MOVEMENTS; HEARING; HEARING LOSS; HUMANS; HYDROXYCHOLESTEROLS; INJECTIONS, SPINAL; MALE; NIEMANN-PICK DISEASE, TYPE C;

EID: 84940877686     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2015.07.001     Document Type: Article

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