Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment-A retrospective chart review

Molecular Genetics and Metabolism

Volumn 116, Issue 1-2, 2015, Pages 53-60

  Vockley, Jerry ^a,b   Marsden, Deborah ^c   McCracken, Elizabeth ^a   DeWard, Stephanie ^a   Barone, Amanda ^a   Hsu, Kristen ^c   Kakkis, Emil ^c  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF PITTSBURGH GRADUATE SCHOOL OF PUBLIC HEALTH   (United States)

^c ULTRAGENYX PHARMACEUTICAL INC   (United States)

Author keywords

Long chain fatty acid oxidation disorder; Retrospective medical chart review; Safety and effectiveness; Triheptanoin

Indexed keywords

LONG CHAIN FATTY ACID; TRIHEPTANOIN; FATTY ACID; TRIACYLGLYCEROL;

ABNORMAL LABORATORY RESULT; ADOLESCENT; ADULT; AGED; ARTICLE; CARDIOMYOPATHY; CHILD; CLINICAL ARTICLE; CLINICAL EVALUATION; CLINICAL FEATURE; COHORT ANALYSIS; COMPASSIONATE USE; DIARRHEA; DISEASE COURSE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; GASTROENTERITIS; GASTROINTESTINAL REFLUX; GASTROINTESTINAL SYMPTOM; HOSPITALIZATION; HUMAN; HYPOGLYCEMIA; INFANT; LENGTH OF STAY; LETHARGY; LONG CHAIN FATTY ACID OXIDATION DISORDER; LOOSE FECES; MALE; MEDICAL RECORD REVIEW; METABOLIC DISORDER; PRESCHOOL CHILD; PRIORITY JOURNAL; RETROSPECTIVE STUDY; RHABDOMYOLYSIS; SCHOOL CHILD; STOMACH PAIN; TREATMENT OUTCOME; VOMITING; CARDIOMYOPATHIES; CRITICAL CARE OUTCOME; LIPID METABOLISM, INBORN ERRORS; METABOLISM; MIDDLE AGED; MORTALITY; OXIDATION REDUCTION REACTION; PATHOLOGY; PATHOPHYSIOLOGY; SURVIVOR; YOUNG ADULT;

ADOLESCENT; ADULT; CARDIOMYOPATHIES; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; CRITICAL CARE OUTCOMES; FATTY ACIDS; FEMALE; HUMANS; HYPOGLYCEMIA; INFANT; LIPID METABOLISM, INBORN ERRORS; MALE; MIDDLE AGED; OXIDATION-REDUCTION; RETROSPECTIVE STUDIES; RHABDOMYOLYSIS; SURVIVORS; TRIGLYCERIDES; YOUNG ADULT;

EID: 84940894295     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2015.06.006     Document Type: Article

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