Composite biomarkers for assessing duchenne muscular dystrophy: An initial assessment

Pediatric Neurology

Volumn 52, Issue 2, 2015, Pages 202-205

  Shklyar, Irina ^a   Pasternak, Amy ^b   Kapur, Kush ^b   Darras, Basil T ^b   Rutkove, Seward B ^a  

^a BETH ISRAEL DEACONESS MEDICAL CENTER   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

Author keywords

biomarker; composite; Duchenne muscular dystrophy; electrical impedance myography; outcome measure; quantitative ultrasound

Indexed keywords

BIOLOGICAL MARKER;

6 MINUTE WALK TEST; ARTICLE; ASSESSMENT OF HUMANS; CHILD; CLINICAL ARTICLE; CLINICAL ASSESSMENT; CONTROLLED STUDY; DIAGNOSTIC TEST ACCURACY STUDY; DISEASE SEVERITY; DUCHENNE MUSCULAR DYSTROPHY; DYNAMOMETRY; ECHOGRAPHY; ELECTRICAL IMPEDANCE MYOGRAPHY; ELECTROMYOGRAPHY; EXPERIMENTAL LOCOMOTOR ACTIVITY TEST; GRAY SCALE ECHOGRAPHY; HUMAN; IMPEDANCE; MALE; MATHEMATICAL ANALYSIS; NORTH STAR AMBULATORY ASSESSMENT; OUTCOME ASSESSMENT; PREDICTIVE VALUE; PRIORITY JOURNAL; QUANTITATIVE DIAGNOSIS; SCHOOL CHILD; SCORING SYSTEM; ADOLESCENT; COMPLICATION; DISEASE COURSE; DYNAMOMETER; FEMALE; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; MYOGRAPHY; PATHOPHYSIOLOGY; PRESCHOOL CHILD; PROCEDURES; SKELETAL MUSCLE; STATISTICS;

ADOLESCENT; BIOMARKERS; CHILD; CHILD, PRESCHOOL; DISEASE PROGRESSION; ELECTRIC IMPEDANCE; FEMALE; HUMANS; MALE; MUSCLE STRENGTH DYNAMOMETER; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOGRAPHY; OUTCOME ASSESSMENT (HEALTH CARE); STATISTICS AS TOPIC;

EID: 84922796260     PISSN: 08878994     EISSN: 18735150     Source Type: Journal    
DOI: 10.1016/j.pediatrneurol.2014.09.014     Document Type: Article

References (23)

1. S. Cirak, L. Feng, and K. Anthony Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy Mol Ther 20 2012 462 467
2. S.F. Nelson, R.H. Crosbie, M.C. Miceli, and M.J. Spencer Emerging genetic therapies to treat Duchenne muscular dystrophy Curr Opin Neurol 22 2009 532 538
3. H. Amthor, and W.M. Hoogaars Interference with myostatin/ActRIIB signaling as a therapeutic strategy for Duchenne muscular dystrophy Curr Gene Ther 12 2012 245 259
4. L. Zhou, and H. Lu Targeting fibrosis in Duchenne muscular dystrophy J Neuropathol Exp Neurol 69 2010 771 776
5. C.M. McDonald, E.K. Henricson, and J.J. Han The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy Muscle Nerve 41 2010 500 510
6. E.S. Mazzone, S. Messina, and G. Vasco Reliability of the North Star Ambulatory Assessment in a multicentric setting Neuromuscul Disord 19 2009 458 461
7. E.P. Hoffman, and E.M. Connor Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies Discov Med 16 2013 233 239
8. E.L. Finanger, B. Russman, S.C. Forbes, W.D. Rooney, G.A. Walter, and K. Vandenborne Use of skeletal muscle MRI in diagnosis and monitoring disease progression in Duchenne muscular dystrophy Phys Med Rehabil Clin N Am 23 2012 1 10
9. S. Pillen, J.P. van Dijk, G. Weijers, W. Raijmann, C.L. de Korte, and M.J. Zwarts Quantitative gray-scale analysis in skeletal muscle ultrasound: a comparison study of two ultrasound devices Muscle Nerve 39 2009 781 786
10. S.B. Rutkove Electrical impedance myography: background, current state, and future directions Muscle Nerve 40 2009 936 946
11. M. Jansen, N. van Alfen, M.W. Nijhuis van der Sanden, J.P. van Dijk, S. Pillen, and I.J. de Groot Quantitative muscle ultrasound is a promising longitudinal follow-up tool in Duchenne muscular dystrophy Neuromuscul Disord 22 2012 306 317
12. J. Li, T.R. Geisbush, G.D. Rosen, J. Lachey, A. Mulivor, and S.B. Rutkove Electrical impedance myography for the in and ex vivo assessment of muscular dystrophy (mdx) mouse muscle Muscle Nerve 49 2014 829 835
13. S.B. Rutkove, T.R. Geisbush, and A. Mijailovic Cross-sectional evaluation of electrical impedance myography and quantitative ultrasound for the assessment of Duchenne muscular dystrophy in a clinical trial setting Pediatr Neurol 51 2014 88 92
14. S.B. Rutkove, and B.T. Darras Electrical impedance myography for the assessment of children with muscular dystrophy: a preliminary study J Phys Conf Ser 434 2013
15. A.H. Poonawalla, S. Datta, and V. Juneja Composite MRI scores improve correlation with EDSS in multiple sclerosis Mult Scler 16 2010 1117 1125
16. G.R. Cutter, M.L. Baier, and R.A. Rudick Development of a multiple sclerosis functional composite as a clinical trial outcome measure Brain 122 Pt 5 1999 871 882
17. P. Narayanaswami, A.J. Spieker, P. Mongiovi, J.C. Keel, S.C. Muzin, and S.B. Rutkove Utilizing a handheld electrode array for localized muscle impedance measurements Muscle Nerve 46 2012 257 263
18. C.M. Zaidman, J. Wu, S. Wilder, B.T. Darras, and S.B. Rutkove Minimal training is required to reliably perform quantitative ultrasound of muscle Muscle Nerve 50 2014 124 128
19. X.L. Meng, R. Rosenthal, and D.B. Rubin Comparing correlation coefficients Psych Bulletin 111 1992 172 175
20. N.G. Faux, C.W. Ritchie, and A. Gunn PBT2 rapidly improves cognition in Alzheimer's Disease: additional phase II analyses J Alzheimers Dis 20 2010 509 516
21. A. Rizk-Jackson, D. Stoffers, and S. Sheldon Evaluating imaging biomarkers for neurodegeneration in pre-symptomatic Huntington's disease using machine learning techniques Neuroimage 56 2011 788 796
22. T. Srivastava, B.T. Darras, J.S. Wu, and S.B. Rutkove Machine learning algorithms to classify spinal muscular atrophy subtypes Neurology 79 2012 358 364
23. FDA. Biomarker qualification context of use. Available at: http://www.fda.gov/drugs/developmentapprovalprocess/drugdevelopmenttoolsqualificationprogram/ucm284620.htm.Accessed May 09, 2014.