UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24 weeks of treatment

Molecular Genetics and Metabolism

Volumn 120, Issue 4, 2017, Pages 370-377

  Vockley, J ^a   Burton, B ^b   Berry, G T ^c   Longo, N ^d   Phillips, J ^e   Sanchez Valle, A ^f   Tanpaiboon, P ^g   Grunewald, S ^h   Murphy, E ⁱ   Humphrey, R ^j   Mayhew, J ^k   Bowden, A ^k   Zhang, L ^k   Cataldo, J ^k   Marsden, D L ^k   Kakkis, E ^k  

^a CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^b CHILDREN'S MEMORIAL HOSPITAL   (United States)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d UNIVERSITY OF UTAH   (United States)

^e VANDERBILT UNIVERSITY MEDICAL CENTER   (United States)

^f UNIVERSITY OF SOUTH FLORIDA   (United States)

^g CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^h UNIVERSITY COLLEGE LONDON   (United Kingdom)

ⁱ NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

^j UNIVERSITY OF MONTANA   (United States)

^k ULTRAGENYX PHARMACEUTICAL INC   (United States)

more..

Author keywords

FAOD; Fatty acid oxidation disorders; LC FAOD; LCHAD; Metabolic disorders; Triheptanoin; UX007; VLCAD

Indexed keywords

TRIHEPTANOIN; FATTY ACID; TRIACYLGLYCEROL;

12 MINUTE WALK TEST; ABDOMINAL PAIN; ACNE; ADOLESCENT; ADULT; ARTICLE; BICYCLE ERGOMETRY; CHILD; CLINICAL ARTICLE; DIARRHEA; DISEASE SEVERITY; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENDURANCE; EXERCISE TEST; EXERCISE TOLERANCE; FEMALE; FEVER; FLATULENCE; GASTROINTESTINAL SYMPTOM; HEADACHE; HUMAN; INBORN ERROR OF METABOLISM; LONG CHAIN FATTY ACID OXIDATION DISORDER; MALE; MULTICENTER STUDY; MUSCLE FUNCTION; MUSCULOSKELETAL DISEASE; NAUSEA; OPEN STUDY; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PROSPECTIVE STUDY; QUALITY OF LIFE; RHABDOMYOLYSIS; SHORT FORM 12; TREATMENT DURATION; UPPER RESPIRATORY TRACT INFECTION; VIRAL GASTROENTERITIS; VOMITING; WALK TEST; CLINICAL TRIAL; DRUG EFFECTS; INFANT; LIPID METABOLISM, INBORN ERRORS; METABOLISM; MIDDLE AGED; NEWBORN; PATHOPHYSIOLOGY; PRESCHOOL CHILD; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; FATTY ACIDS; FEMALE; HUMANS; INFANT; INFANT, NEWBORN; LIPID METABOLISM, INBORN ERRORS; MALE; MIDDLE AGED; PHYSICAL ENDURANCE; PROSPECTIVE STUDIES; QUALITY OF LIFE; TREATMENT OUTCOME; TRIGLYCERIDES; WALK TEST; YOUNG ADULT;

EID: 85011891317     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2017.02.005     Document Type: Article

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