A safety trial of high dose glyceryl triacetate for Canavan disease

Molecular Genetics and Metabolism

Volumn 103, Issue 3, 2011, Pages 203-206

  Segel, Reeval ^a,b   Anikster, Yair ^c,d   Zevin, Shoshana ^a   Steinberg, Avraham ^a   Gahl, William A ^e   Fisher, Drora ^a,b   Staretz Chacham, Orna ^f   Zimran, Ari ^a,b   Altarescu, Gheona ^a,b  

^a SHAARE ZEDEK MEDICAL CENTER   (Israel)

^b HEBREW UNIVERSITY   (Israel)

^c SHEBA MEDICAL CENTER   (Israel)

^d TEL AVIV UNIVERSITY   (Israel)

^e NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^f BEN GURION UNIVERSITY OF THE NEGEV   (Israel)

Author keywords

ASPA; Aspartoacylase deficiency; Canavan disease; Glyceryltriacetate; GTA; Safety

Indexed keywords

ALANINE AMINOTRANSFERASE; ALBUMIN; ALKALINE PHOSPHATASE; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; CALCIUM; CHLORIDE; CHOLESTEROL; CREATININE; GLUCOSE; HEMOGLOBIN; LACTATE DEHYDROGENASE; N ACETYLASPARTIC ACID; OMEPRAZOLE; PHOSPHORUS; POTASSIUM; PROTEIN; SODIUM; TRIACETIN; UREA;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALBUMIN BLOOD LEVEL; ALKALINE PHOSPHATASE BLOOD LEVEL; AMINO ACID BLOOD LEVEL; ARTICLE; ARTIFICIAL MILK; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BILIRUBIN BLOOD LEVEL; CALCIUM BLOOD LEVEL; CANAVAN DISEASE; CASE REPORT; CHLORIDE BLOOD LEVEL; CHOLESTEROL BLOOD LEVEL; CREATININE BLOOD LEVEL; DRUG DOSE INCREASE; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; ERYTHROCYTE; GENE MUTATION; GLUCOSE BLOOD LEVEL; HEMATOCRIT; HEMOGLOBIN BLOOD LEVEL; HUMAN; INFANT; LACTATE DEHYDROGENASE BLOOD LEVEL; LEUKOCYTE; LOW DRUG DOSE; LYMPHOCYTE; MEAN CORPUSCULAR HEMOGLOBIN; MEAN CORPUSCULAR VOLUME; MONOCYTE; MOTOR PERFORMANCE; NEUTROPHIL; PHOSPHATE BLOOD LEVEL; POTASSIUM BLOOD LEVEL; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SIDE EFFECT; SODIUM BLOOD LEVEL; STOMACH DISCOMFORT; THROMBOCYTE; UREA BLOOD LEVEL;

BRAIN; CANAVAN DISEASE; FEMALE; HUMANS; INFANT; MAGNETIC RESONANCE IMAGING; MALE; NEUROPROTECTIVE AGENTS; TREATMENT OUTCOME; TRIACETIN;

MUS;

EID: 79958041841     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.03.012     Document Type: Article

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