One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

Pediatric Neurology

Volumn 50, Issue 6, 2014, Pages 557-563

  Connolly, Anne M ^a,b   Florence, Julaine M ^a   Cradock, Mary M ^b   Eagle, Michelle ^h,i   Flanigan, Kevin M ^c,d   McDonald, Craig M ^e   Karachunski, Peter I ^f   Darras, Basil T ^g   Bushby, Kate ^h,i   Malkus, Elizabeth C ^a   Golumbek, Paul T ^a,b   Zaidman, Craig M ^a,b   Miller, J Philip ^a   Mendell, Jerry R ^c,d  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b ST LOUIS CHILDREN S HOSPITAL   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d NATIONWIDE CHILDREN S HOSPITAL   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f UNIVERSITY OF MINNESOTA   (United States)

^g BOSTON CHILDREN S HOSPITAL   (United States)

^h NEWCASTLE UNIVERSITY   (United Kingdom)

ⁱ NEWCASTLE UNIVERSITY   (United Kingdom)

Author keywords

Bayley III; clinical trial outcomes; Duchenne muscular dystrophy; infant development

Indexed keywords

ADAPTIVE BEHAVIOR; ARTICLE; BAYLEY SCALES OF INFANT DEVELOPMENT; CLINICAL ARTICLE; CLINICAL EVALUATION; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EXON; FAMILY HISTORY; GENE DELETION; GENE DISRUPTION; HUMAN; INFANT; MALE; MOTOR DEVELOPMENT; MOTOR PERFORMANCE; NONSENSE MUTATION; OUTCOME ASSESSMENT; PRIORITY JOURNAL; CHILD DEVELOPMENT; CLINICAL TRIAL; COGNITION; FOLLOW UP; GENETICS; LANGUAGE; LANGUAGE TEST; LONGITUDINAL STUDY; MOTOR ACTIVITY; MULTICENTER STUDY; MUSCULAR DYSTROPHY, DUCHENNE; PATHOPHYSIOLOGY; PRESCHOOL CHILD; PSYCHOLOGY;

CHILD DEVELOPMENT; CHILD, PRESCHOOL; COGNITION; FOLLOW-UP STUDIES; HUMANS; INFANT; LANGUAGE; LANGUAGE TESTS; LONGITUDINAL STUDIES; MALE; MOTOR ACTIVITY; MOTOR SKILLS; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 84901030899     PISSN: 08878994     EISSN: 18735150     Source Type: Journal    
DOI: 10.1016/j.pediatrneurol.2014.02.006     Document Type: Article

References (28)

1. L. Merlini, D. Dell'Accio, and C. Granata Reliability of dynamic strength knee muscle testing in children J Orthop Sports Phys Ther 22 1995 73 76
2. C.M. McDonald, R.T. Abresch, and G.T. Carter et al. Profiles of neuromuscular diseases. Duchenne muscular dystrophy Am J Phys Med Rehabil 74 5 Suppl 1995 S70 S92
3. M.H. Brooke, G.M. Fenichel, and R.C. Griggs et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy Neurology 39 1989 475 481
4. M. Brooke, G.M. Fenichel, and R.C. Griggs et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history Muscle Nerve 6 1983 91 103
5. J.E. Mayhew, J.M. Florence, and T.P. Mayhew et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy Muscle Nerve 35 2007 36 42
6. C.M. McDonald, E.K. Henricson, and J.J. Han et al. The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations Muscle Nerve 42 2010 966 974
7. E. Mazzone, G. Vasco, and M.P. Sormani et al. Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study Neurology 77 2011 250 256
8. R.A. Smith, R.G. Newcombe, J.R. Sibert, and P.S. Harper Assessment of locomotor function in young boys with Duchenne muscular dystrophy Muscle Nerve 14 1991 462 469
9. M. Pane, R. Scalise, and A. Berardinelli et al. Early neurodevelopmental assessment in Duchenne muscular dystrophy Neuromuscul Disord 23 2013 451 455
10. A.M. Connolly, J.M. Florence, and M.M. Cradock et al. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network Neuromuscul Disord 23 2013 529 539
11. J.R. Mendell, C. Shilling, and N.D. Leslie et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy Ann Neurol 71 2012 304 313
12. N. Bayley Bayley Scales of Infant and Toddler Development 3rd ed. 2005 Harcourt Assessment San Antonio
13. S.I. Greenspan Clinical assessment of emotional milestones in infancy and early childhood Pediatr Clin North Am 38 1991 1371 1385
14. F. Faul, E. Erdfelder, A. Buchner, and A.G. Lang Statistical power analyses using G*Power 3.1: tests for correlation and regression analyses Behav Res Methods 41 2009 1149 1160
15. K.M. Flanigan, D.M. Dunn, and A. von Niederhausern et al. Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort Hum Mutat 30 2009 1657 1666
16. A. Govoni, F. Magri, and S. Brajkovic et al. Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy Cell Mol Life Sci 70 2013 4584 4602
17. T. Koo, and M.J. Wood Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy Hum Gene Ther 24 2013 479 488
18. C.M. McDonald, E.K. Henricson, and R.T. Abresch et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study Muscle Nerve 48 2013 343 356
19. J.R. Mendell, L. Rodino-Klapac, and Z. Sahenk et al. Gene therapy for muscular dystrophy: lessons learned and path forward Neurosci Lett 527 2012 90 99
20. R.S. Finkel Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: aminoglycosides and ataluren (PTC124) J Child Neurol 25 2010 1158 1164
21. K.D. Huebner, D.S. Jassal, O. Halevy, M. Pines, and J.E. Anderson Functional resolution of fibrosis in mdx mouse dystrophic heart and skeletal muscle by halofuginone Am J Physiol Heart Circ Physiol 294 2008 H1550 H1561
22. E.A. Martin, R. Barresi, and B.J. Byrne et al. Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy Sci Transl Med 4 2012 162ra155
23. A. Asai, N. Sahani, M. Kaneki, Y. Ouchi, J.A. Martyn, and S.E. Yasuhara Primary role of functional ischemia, quantitative evidence for the two-hit mechanism, and phosphodiesterase-5 inhibitor therapy in mouse muscular dystrophy PLoS One 2 2007 e806
24. K.M. Flanigan, E. Ceco, and K.M. Lamar et al. LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy Ann Neurol 73 2013 481 488
25. E. Pegoraro, E.P. Hoffman, and L. Piva et al. SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy Neurology 76 2011 219 226
26. J.R. Mendell, and M. Lloyd-Puryear Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy Muscle Nerve 48 2013 21 26
27. A. Spittle, J. Orton, P. Anderson, R. Boyd, and L.W. Doyle Early developmental intervention programmes post-hospital discharge to prevent motor and cognitive impairments in preterm infants Cochrane Database Syst Rev 2012 CD005495
28. C.H. Blauw-Hospers, V.B. de Graaf-Peters, T. Dirks, A.F. Bos, and M. Hadders-Algra Does early intervention in infants at high risk for a developmental motor disorder improve motor and cognitive development? Neurosci Biobehav Rev 31 2007 1201 1212