The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: Considerations for the design of clinical trials

Journal of Neurology, Neurosurgery and Psychiatry

Volumn 87, Issue 2, 2016, Pages 149-155

  Ricotti, Valeria ^a,b   Ridout, Deborah A ^a,b   Pane, Marika ^c   Main, Marion ^a,b   Mayhew, Anna ^d   Mercuri, Eugenio ^a,c   Manzur, Adnan Y ^a,b   Muntoni, Francesco ^a,b   Robb, S ^b   Quinlivan, R ^b   Sarkozy, A ^b   Butler, J ^b   Bushby, K ^d   Straub, V ^d   Guglieri, M ^d   Eagle, M ^d   Roper, H ^e   McMurchie, H ^e   Childs, A ^f   Pysden, K ^f   Pallant, L ^f   Spinty, S ^g   Peachey, G ^g   Shillington, A ^g   Wraige, E ^h   Jungbluth, H ^h   Sheehan, J ^h   Spahr, R ^h   Hughes, I ⁱ   Bateman, E ⁱ   Cammiss, C ⁱ   Willis, T ^j   Groves, L ^j   Emery, N ^j   Baxter, P ^k   Senior, M ^k   Scott, E ^k   Hartley, L ^l   Parsons, B ^l   Majumdar, A ^m   Jenkins, L ^m   Toms, B ^m   Naismith, K ⁿ   Keddie, A ⁿ   Horrocks, I ^o   Di Marco, M ^o   Chow, G ^p   Miah, A ^p   De Goede, C ^q   Thomas, N ^r   Geary, M ^r   Palmer, J ^r   White, C ^s   Greenfield, K ^s   Wilson, I ^b   more..

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^c CATHOLIC UNIVERSITY   (Italy)

^d NEWCASTLE UNIVERSITY   (United Kingdom)

^e BIRMINGHAM HEARTLANDS HOSPITAL   (United Kingdom)

^f LEEDS GENERAL INFIRMARY   (United Kingdom)

^g ALDER HEY CHILDREN S HOSPITAL   (United Kingdom)

^h ST THOMAS HOSPITAL   (United Kingdom)

ⁱ ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^j ROBERT JONES AND AGNES HUNT ORTHOPAEDIC HOSPITAL   (United Kingdom)

^k SHEFFIELD CHILDREN S HOSPITAL   (United Kingdom)

^l UNIVERSITY HOSPITAL OF WALES   (United Kingdom)

^m FRENCHAY HOSPITAL   (United Kingdom)

ⁿ King's Cross Hospital   (United Kingdom)

^o ROYAL HOSPITAL FOR SICK CHILDREN   (United Kingdom)

^p QUEEN S MEDICAL CENTRE   (United Kingdom)

^q ROYAL PRESTON HOSPITAL   (United Kingdom)

^r SOUTHAMPTON GENERAL HOSPITAL   (United Kingdom)

^s MORRISTON HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

GLUCOCORTICOID; ANTIINFLAMMATORY AGENT;

ADOLESCENT; ARTICLE; CHILD; CLINICAL STUDY; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; GENE DELETION; GENE MUTATION; GENOTYPE; HUMAN; ITALIAN (CITIZEN); MAJOR CLINICAL STUDY; MALE; NORTHSTAR AMBULATORY ASSESSMENT SCORE; PRIORITY JOURNAL; SCORING SYSTEM; UNITED KINGDOM; CLINICAL TRIAL (TOPIC); COHORT ANALYSIS; DISEASE COURSE; EARLY DIAGNOSIS; FOLLOW UP; GENETICS; ITALY; METHODOLOGY; MUSCULAR DYSTROPHY, DUCHENNE; ONSET AGE; STANDARDS; WALKING; YOUNG ADULT;

ADOLESCENT; AGE OF ONSET; ANTI-INFLAMMATORY AGENTS; CHILD; CLINICAL TRIALS AS TOPIC; COHORT STUDIES; DISEASE PROGRESSION; EARLY DIAGNOSIS; EXONS; FOLLOW-UP STUDIES; GENE DELETION; GENOTYPE; GLUCOCORTICOIDS; HUMANS; ITALY; MALE; MUSCULAR DYSTROPHY, DUCHENNE; RESEARCH DESIGN; UNITED KINGDOM; WALKING; YOUNG ADULT;

EID: 84958863314     PISSN: 00223050     EISSN: 1468330X     Source Type: Journal    
DOI: 10.1136/jnnp-2014-309405     Document Type: Article

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