Quality of life is improved and kidney function preserved in patients with nephropathic cystinosis treated for 2 years with delayed-release cysteamine bitartrate

Journal of Pediatrics

Volumn 165, Issue 3, 2014, Pages 528-533.e1

  Langman, Craig B ^a   Greenbaum, Larry A ^b   Grimm, Paul ^c   Sarwal, Minnie ^c   Niaudet, Patrick ^d   Deschenes, Georges ^e   Cornelissen, Elisabeth A M ^f   Morin, Denis ^g   Cochat, Pierre ^h   Elenberg, Ewa ⁱ   Hanna, Christian ^a   Gaillard, Segolene ^j   Bagger, Mary Jo ^k   Rioux, Patrice ^k  

^a NORTHWESTERN UNIVERSITY   (United States)

^b EMORY UNIVERSITY   (United States)

^c STANFORD UNIVERSITY MEDICAL CENTER   (United States)

^d HÔPITAL NECKER ENFANTS MALADES   (France)

^e HÔPITAL ROBERT DEBRÉ   (France)

^f RADBOUD UNIVERSITY NIJMEGEN   (Netherlands)

^g MONTPELLIER UNIVERSITY HOSPITAL   (France)

^h HOSPICES CIVILS DE LYON   (France)

ⁱ TEXAS CHILDREN S HOSPITAL   (United States)

^j INSERM   (France)

^k Raptor Pharmaceutical Inc   (United States)

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Author keywords

[No Author keywords available]

Indexed keywords

CYSTINE; MERCAPTAMINE; CYSTINE DEPLETING AGENT; DELAYED RELEASE FORMULATION;

ARTICLE; BODY GROWTH; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CYSTINOSIS; DELAYED RELEASE FORMULATION; DIARRHEA; DRUG EFFECT; DRUG EFFICACY; FEMALE; GASTROINTESTINAL DISEASE; GLOMERULUS FILTRATION RATE; GROWTH RATE; HEADACHE; HUMAN; KIDNEY FUNCTION; LEUKOCYTE; MALE; NAMED INVENTORIES, QUESTIONNAIRES AND RATING SCALES; NEPHROPATHIC CYSTINOSIS; PEDIATRIC QUALITY OF LIFE INVENTORY; PRIORITY JOURNAL; QUALITY OF LIFE; RESPIRATORY TRACT DISEASE; SOCIAL STATUS; VOMITING; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; PATHOPHYSIOLOGY; PROSPECTIVE STUDY; TIME;

CHILD; CYSTEAMINE; CYSTINE DEPLETING AGENTS; CYSTINOSIS; DELAYED-ACTION PREPARATIONS; FEMALE; GLOMERULAR FILTRATION RATE; HUMANS; MALE; PROSPECTIVE STUDIES; QUALITY OF LIFE; TIME FACTORS;

EID: 84906794208     PISSN: 00223476     EISSN: 10976833     Source Type: Journal    
DOI: 10.1016/j.jpeds.2014.05.013     Document Type: Article

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