Safety and proof-of-concept study of oral QLT091001 in retinitis pigmentosa due to inherited deficiencies of retinal pigment epithelial 65 protein (RPE65) or lecithin: Retinol acyltransferase (LRAT)

PLoS ONE

Volumn 10, Issue 12, 2015, Pages

  Scholl, Hendrik P N ^a   Moore, Anthony T ^b,c   Koenekoop, Robert K ^d   Wen, Yuquan ^e,f   Fishman, Gerald A ^g   Van Den Born, L Ingeborgh ^h   Bittner, Ava ^a,i   Bowles, Kristen ^a,j   Fletcher, Emily C ^a,k   Collison, Frederick T ^g   Dagnelie, Gislin ^a   Eposti, Simona Degli ^b   Michaelides, Michel ^b   Saperstein, David A ^l,m   Schuchard, Ronald A ^m,n   Barnes, Claire ^m,o   Zein, Wadih ^p   Zobor, Ditta ^q   Birch, David G ^e,r   Mendola, Janine D ^d   Zrenner, Eberhart ^q   more..

^a JOHNS HOPKINS UNIVERSITY   (United States)

^b MOORFIELDS EYE HOSPITAL   (United Kingdom)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d MCGILL UNIVERSITY HEALTH CENTRE   (Canada)

^e RETINA FOUNDATION OF THE SOUTHWEST   (United States)

^f BAYLOR UNIVERSITY MEDICAL CENTER   (United States)

^g Chicago Lighthouse   (United States)

^h ROTTERDAM EYE HOSPITAL   (Netherlands)

ⁱ NOVA SOUTHEASTERN UNIVERSITY   (United States)

^j UNIVERSITY OF HOUSTON   (United States)

^k Gloucester Royal Hospitals   (United States)

^l Vitreoretinal of Washington   (United States)

^m QLT Inc   (United States)

ⁿ STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^o VA PALO ALTO HEALTH CARE SYSTEM   (United States)

^p NATIONAL EYE INSTITUTE   (United States)

^q UNIVERSITY OF TÜBINGEN   (Germany)

^r UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

9 CIS RETINOL ACETATE; ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; BICARBONATE; HIGH DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN; PHOSPHATIDYLCHOLINE; POTASSIUM; RETINAL PIGMENT EPITHELIAL 65 PROTEIN; RETINOL ACYLTRANSFERASE; THYROXINE; TRIACYLGLYCEROL; UNCLASSIFIED DRUG; VISUAL PIGMENT; ACYLTRANSFERASE; ANTINEOPLASTIC AGENT; CIS TRANS ISOMERASE; LECITHIN-RETINOL ACYLTRANSFERASE; RETINOID ISOMEROHYDROLASE; RETINOL; RETINOL ACETATE;

ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BICARBONATE BLOOD LEVEL; CHILD; CLINICAL ARTICLE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; FEMALE; FREE THYROXINE INDEX; FUNCTIONAL MAGNETIC RESONANCE IMAGING; GENE; GENE MUTATION; GENETIC DISORDER; HEADACHE; HEMATOCRIT; HUMAN; LEUKOCYTE COUNT; LIPOPROTEIN BLOOD LEVEL; LRAT GENE; MALE; MIDDLE AGED; MULTICENTER STUDY; NAUSEA; OPEN STUDY; OPTICAL COHERENCE TOMOGRAPHY; OUTCOME ASSESSMENT; PHOTOPHOBIA; PHOTORECEPTOR; POTASSIUM BLOOD LEVEL; PRESCHOOL CHILD; RETINAL THICKNESS; RETINITIS PIGMENTOSA; RPE65 GENE; SCHOOL CHILD; SIDE EFFECT; STRUCTURE ACTIVITY RELATION; TRIACYLGLYCEROL BLOOD LEVEL; VISUAL ACUITY; VISUAL FIELD; VOMITING; YOUNG ADULT; ANALOGS AND DERIVATIVES; BRAIN CORTEX; CLINICAL TRIAL; DIAGNOSTIC IMAGING; DOSE CALCULATION; DRUG EFFECTS; GENETICS; METABOLISM; ORAL DRUG ADMINISTRATION; PATHOLOGY; RADIOGRAPHY; RETINA GANGLION CELL; RETINA NERVE CELL; SINGLE NUCLEOTIDE POLYMORPHISM; TREATMENT OUTCOME;

ACYLTRANSFERASES; ADMINISTRATION, ORAL; ADULT; ANTICARCINOGENIC AGENTS; CEREBRAL CORTEX; CHILD; CIS-TRANS-ISOMERASES; DRUG DOSAGE CALCULATIONS; FEMALE; HUMANS; MALE; MIDDLE AGED; POLYMORPHISM, SINGLE NUCLEOTIDE; RADIOGRAPHY; RETINAL GANGLION CELLS; RETINAL NEURONS; RETINITIS PIGMENTOSA; TREATMENT OUTCOME; VISUAL ACUITY; VISUAL FIELDS; VITAMIN A; YOUNG ADULT;

EID: 84955599907     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0143846     Document Type: Article

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