Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

Journal of Medical Genetics

Volumn 54, Issue 4, 2017, Pages 288-296

  Hughes, Derralynn A ^a   Nicholls, Kathleen ^b   Shankar, Suma P ^c   Sunder Plassmann, Gere ^d   Koeller, David ^e   Nedd, Khan ^f   Vockley, Gerard ^g   Hamazaki, Takashi ^f,h   Lachmann, Robin ⁱ   Ohashi, Toya ^j   Olivotto, Iacopo ^k   Sakai, Norio ^h   Deegan, Patrick ^l   Dimmock, David ^m   Eyskens, François ⁿ   Germain, Dominique P ^o   Goker Alpan, Ozlem ^p   Hachulla, Eric ^q   Jovanovic, Ana ^r   Lourenco, Charles M ^s   Narita, Ichiei ^t   Thomas, Mark ^u   Wilcox, William R ^v   Bichet, Daniel G ^w   Schiffmann, Raphael ^x   Ludington, Elizabeth ^y   Viereck, Christopher ^z   Kirk, John ^z   Yu, Julie ^z   Johnson, Franklin ^z   Boudes, Pol ^aa   Benjamin, Elfrida R ^z   Lockhart, David J ^ab   Barlow, Carrolee ^ac   Skuban, Nina ^z   Castelli, Jeffrey P ^z   Barth, Jay ^z   Feldt Rasmussen, Ulla ^ad   more..

^a ROYAL FREE LONDON NHS FOUNDATION TRUST   (United Kingdom)

^b ROYAL MELBOURNE HOSPITAL   (Australia)

^c EMORY UNIVERSITY   (United States)

^d MEDICAL UNIVERSITY OF VIENNA   (Austria)

^e OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^f Infusion Associates   (United States)

^g CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^h OSAKA CITY UNIVERSITY HOSPITAL   (Japan)

ⁱ NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

^j JIKEI UNIVERSITY HOSPITAL   (Japan)

^k CAREGGI UNIVERSITY HOSPITAL   (Italy)

^l ADDENBROOKE S HOSPITAL   (United Kingdom)

^m Addenbrooke's Hospital ^*  (United States)

ⁿ Univeritair Ziekenhaus Anterpen   (Belgium)

^o LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^p O and O Alpan LLC   (United States)

^q LILLE UNIVERSITY HOSPITAL   (France)

^r SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^s UNIVERSITY OF SÃO PAULO   (Brazil)

^t NIIGATA UNIVERSITY GRADUATE SCHOOL OF MEDICAL AND DENTAL SCIENCES   (Japan)

^u ROYAL PERTH HOSPITAL   (Australia)

^v EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^w HÔPITAL DU SACRÉ COEUR DE MONTRÉAL   (Canada)

^x BAYLOR UNIVERSITY MEDICAL CENTER   (United States)

^y Agility Clinical Inc   (United States)

^z Amicus Therapeutics Inc   (United States)

^aa CymaBay Therapeutics Inc   (United States)

^ab TranscripTx   (United States)

^ac PARKINSON S INSTITUTE   (United States)

^ad Rigshospitalet   (Denmark)

more..

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; GLOBOTRIAOSYLSPHINGOSINE; MIGALASTAT; SPHINGOSINE DERIVATIVE; UNCLASSIFIED DRUG; 1 DEOXYNOJIRIMYCIN; CHAPERONE;

ADULT; AGED; AMINO ACID SUBSTITUTION; ARTICLE; CEREBROVASCULAR DISEASE; CLINICAL OUTCOME; CONTROLLED STUDY; DISEASE COURSE; DISEASE SEVERITY; DRUG EFFECT; DRUG SAFETY; DRUG SUBSTITUTION; DRUG TOLERABILITY; DRUG WITHDRAWAL; ECHOCARDIOGRAPHY; ENZYME REPLACEMENT; FABRY DISEASE; FEMALE; GENE MUTATION; HEADACHE; HEART DISEASE; HEART LEFT VENTRICLE MASS; HUMAN; KIDNEY DISEASE; KIDNEY FUNCTION; MAJOR CLINICAL STUDY; MALE; MONOTHERAPY; OPEN STUDY; PHASE 3 CLINICAL TRIAL; PHENOTYPE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RHINOPHARYNGITIS; TREATMENT DURATION; TREATMENT RESPONSE; ADOLESCENT; ANALOGS AND DERIVATIVES; CLINICAL TRIAL; GENETICS; LYSOSOME; METABOLISM; MIDDLE AGED; ORAL DRUG ADMINISTRATION; PATHOLOGY; PATHOPHYSIOLOGY; TREATMENT OUTCOME;

1-DEOXYNOJIRIMYCIN; ADMINISTRATION, ORAL; ADOLESCENT; ADULT; AGED; ALPHA-GALACTOSIDASE; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FEMALE; HUMANS; LYSOSOMES; MALE; MIDDLE AGED; MOLECULAR CHAPERONES; TREATMENT OUTCOME;

EID: 84995530518     PISSN: 00222593     EISSN: 14686244     Source Type: Journal    
DOI: 10.1136/jmedgenet-2016-104178     Document Type: Article

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