Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: A prospective cohort study

The Lancet

Volumn 386, Issue 10007, 2015, Pages 1955-1963

  Schwahn, Bernd C ^a,h   Van Spronsen, Francjan J ^b   Belaidi, Abdel A ^c,e   Bowhay, Stephen ^a   Christodoulou, John ^f   Derks, Terry G ^b   Hennermann, Julia B ^g   Jameson, Elisabeth ^h   König, Kai ⁱ   McGregor, Tracy L ^j   Font Montgomery, Esperanza ^k   Santamaria Araujo, José A ^l   Santra, Saikat ^m   Vaidya, Mamta ^l,n   Vierzig, Anne ^d   Wassmer, Evangeline ^m   Weis, Ilona ^o   Wong, Flora Y ^p   Veldman, Alex ^p   Schwarz, Günter ^c,l  

^a ROYAL HOSPITAL FOR SICK CHILDREN   (United Kingdom)

^b UNIVERSITY OF GRONINGEN   (Netherlands)

^c UNIVERSITY OF COLOGNE   (Germany)

^d UNIVERSITY HOSPITAL OF COLOGNE   (Germany)

^e UNIVERSITY OF MELBOURNE   (Australia)

^f UNIVERSITY OF SYDNEY   (Australia)

^g JOHANNES GUTENBERG UNIVERSITY   (Germany)

^h MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

ⁱ MERCY HOSPITAL FOR WOMEN   (Australia)

^j MONROE CARELL JR CHILDREN S HOSPITAL AT VANDERBILT   (United States)

^k Children's Hospital of Wisconsin   (United States)

^l Orphatec Pharmaceuticals GmbH   (Germany)

^m BIRMINGHAM CHILDREN S HOSPITAL   (United Kingdom)

ⁿ ROYAL LONDON HOSPITAL   (United Kingdom)

^o Gemeinschaftsklinikum Koblenz Mayen   (Germany)

^p MONASH UNIVERSITY   (Australia)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CYCLIC PYRANOPTERIN PHOSPHATE; CYSTEINE; DNA; MOLYBDENUM COMPLEX; S SULPHOCYSTEINE; UNCLASSIFIED DRUG; URATE; XANTHINE; ORGANOPHOSPHORUS COMPOUND; PTERIN DERIVATIVE;

ARTICLE; BRAIN DISEASE; CLINICAL ARTICLE; COHORT ANALYSIS; COMPASSIONATE USE; CONVULSION; DISEASE MARKER; DRUG EFFICACY; DRUG SAFETY; DRUG SUBSTITUTION; FEMALE; GENERAL CONDITION IMPROVEMENT; HOSPITAL ADMISSION; HUMAN; HUMAN CELL; INFANT; MALE; MOLYBDENUM COFACTOR DEFICIENCY TYPE A; MOLYBDENUM COFACTOR DEFICIENCY TYPE B; MULTICENTER STUDY; NEWBORN; OBSERVATIONAL STUDY; PRIORITY JOURNAL; PROSPECTIVE STUDY; DRUG ADMINISTRATION; METAL METABOLISM, INBORN ERRORS; TREATMENT OUTCOME;

COHORT STUDIES; COMPASSIONATE USE TRIALS; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; INFANT, NEWBORN; MALE; METAL METABOLISM, INBORN ERRORS; ORGANOPHOSPHORUS COMPOUNDS; PTERINS; TREATMENT OUTCOME;

EID: 84947486885     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(15)00124-5     Document Type: Article

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