Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database

Journal of Neuromuscular Diseases

Volumn 4, Issue 4, 2017, Pages 293-306

  Koeks, Zaïda ^y   Bladen, Catherine L ^a   Salgado, David ^ar   Van Zwet, Erik ^y   Pogoryelova, Oksana ^a   McMacken, Grace ^a   Monges, Soledad ^b   Foncuberta, Maria E ^b   Kekou, Kyriaki ^m   Kosma, Konstantina ^m   Dawkins, Hugh ^c   Lamont, Leanne ^c   Bellgard, Matthew I ^d   Roy, Anna J ^e   Chamova, Teodora ^f   Guergueltcheva, Velina ^g   Chan, Sophelia ^l   Korngut, Lawrence ^h   Campbell, Craig ⁱ   Dai, Yi ^j   Wang, Jen ^k   Barišić, Nina ⁿ   Brabec, Petr ^o   Lähdetie, Jaana ^p   Walter, Maggie C ^q   Schreiber Katz, Olivia ^q   Karcagi, Veronika ^r   Garami, Marta ^r   Herczegfalvi, Agnes ^s   Viswanathan, Venkatarman ^t   Bayat, Farhad ^u   Buccella, Filippo ^v   Ferlini, Alessandra ^w   Kimura, En ^x   Van Den Bergen, Janneke C ^y   Rodrigues, Miriam ^z   Roxburgh, Richard ^z   Lusakowska, Anna ^aa   Kostera Pruszczyk, Anna ^aa   Santos, Rosário ^ab   Neagu, Elena ^ac   Artemieva, Svetlana ^ad   Rasic, Vedrana Milic ^ae   Vojinovic, Dina ^ae,af   Posada, Manuel ^ag   Bloetzer, Clemens ^ah   Klein, Andrea ^ai   Díaz Manera, Jordi ^aj   Gallardo, Eduard ^ai   Karaduman, A Ayşe ^ak   Oznur, Tunca ^ak   Topalolu, Haluk ^ak   El Sherif, Rasha ^al   Stringer, Angela ^am   Shatillo, Andriy V ^an   Martin, Ann S ^ao   Peay, Holly L ^ao   Kirschner, Jan ^ap   Flanigan, Kevin M ^aq   Straub, Volker ^a   Bushby, Kate ^a   Béroud, Christophe ^ar   Verschuuren, Jan J ^y   Lochmüller, Hanns ^a   more..

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b HOSPITAL DE PEDIATRÍA PROF DR JUAN P GARRAHAN   (Argentina)

^c DEPARTMENT OF HEALTH   (Australia)

^d MURDOCH UNIVERSITY   (Australia)

^e SCIENTIFIC INSTITUTE OF PUBLIC HEALTH   (Belgium)

^f MEDICAL UNIVERSITY OF SOFIA   (Bulgaria)

^g UNIVERSITY HOSPITAL ALEXANDROVSKA   (Bulgaria)

^h UNIVERSITY OF CALGARY   (Canada)

ⁱ UNIVERSITY OF WESTERN ONTARIO   (Canada)

^j PEKING UNION MEDICAL COLLEGE HOSPITAL   (China)

^k China DMD Care and Support Association C o China Dolls   (China)

^l UNIVERSITY OF HONG KONG   (Hong Kong)

^m UNIVERSITY OF ATHENS MEDICAL SCHOOL   (Greece)

ⁿ UNIVERSITY OF ZAGREB   (Croatia)

^o MASARYK UNIVERSITY   (Czech Republic)

^p TURKU UNIVERSITY HOSPITAL   (Finland)

^q LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^r NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH   (Hungary)

^s SEMMELWEIS UNIVERSITY OF MEDICINE   (Hungary)

^t Kanchi Kamakoti Trust Hospital   (India)

^u PASTEUR INSTITUTE OF IRAN   (Iran)

^v Parent Project Onlus   (Italy)

^w UNIVERSITY OF FERRARA   (Italy)

^x Department of Neurology ^*  (Japan)

^y LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^z AUCKLAND DISTRICT HEALTH BOARD   (New Zealand)

^aa MEDICAL UNIVERSITY OF WARSAW   (Poland)

^ab UP   (Portugal)

^ac NATIONAL INSTITUTE OF LEGAL MEDICINE MINA MINOVICI   (Romania)

^ad Institute of Pediatrics   (Russian Federation)

^ae UNIVERSITY OF BELGRADE   (Serbia)

^af ERASMUS MEDICAL CENTER   (Netherlands)

^ag INSTITUTO DE SALUD CARLOS III   (Spain)

^ah UNIVERSITY OF BERN   (Switzerland)

^ai LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

^aj HOSPITAL DE LA SANTA CREU I SANT PAU   (Spain)

^ak HACETTEPE UNIVERSITY   (Turkey)

^al AIN SHAMS UNIVERSITY   (Egypt)

^am Action Duchenne   (United Kingdom)

^an Inst Neurol Psychiat Narcology   (Ukraine)

^ao Duchenne Connect   (United States)

^ap UNIVERSITY OF FREIBURG   (Germany)

^aq NATIONWIDE CHILDREN S HOSPITAL   (United States)

^ar TIMONE HOSPITAL   (France)

more..

Author keywords

DMD; Duchenne muscular dystrophy; Neuromuscular diseases; TREAT NMD

Indexed keywords

CORTICOSTEROID;

ARTICLE; ASSISTED VENTILATION; CARDIOMYOPATHY; CHILD; COHORT ANALYSIS; CONTROLLED STUDY; CROSS-SECTIONAL STUDY; DATA BASE; DISEASE REGISTRY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; ECHOCARDIOGRAPHY; FEMALE; GENE IDENTIFICATION; GENE MUTATION; GENETIC IDENTIFICATION; GOOD CLINICAL PRACTICE; HEALTH CARE SYSTEM; HEART PROTECTION; HUMAN; MAJOR CLINICAL STUDY; MALE; MUSCLE WEAKNESS; OUTCOME ASSESSMENT; PRESCHOOL CHILD; PRIORITY JOURNAL; RESPIRATORY FAILURE; SCOLIOSIS; SURVIVAL RATE; ADOLESCENT; ADULT; GENETICS; INFANT; NEWBORN; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADRENAL CORTEX HORMONES; ADULT; CHILD; CHILD, PRESCHOOL; CROSS-SECTIONAL STUDIES; DATABASES AS TOPIC; HUMANS; INFANT; INFANT, NEWBORN; MALE; MUSCULAR DYSTROPHY, DUCHENNE; TREATMENT OUTCOME; YOUNG ADULT;

EID: 85035064593     PISSN: 22143599     EISSN: 22143602     Source Type: Journal    
DOI: 10.3233/JND-170280     Document Type: Article

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