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723 results on '"McDonald, Craig M."'

217. Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study.

Author

Bello, Luca, Kesari, Akanchha, Gordish‐Dressman, Heather, Cnaan, Avital, Morgenroth, Lauren P., Punetha, Jaya, Duong, Tina, Henricson, Erik K., Pegoraro, Elena, McDonald, Craig M., and Hoffman, Eric P.

Abstract

Objective We studied the effects of LTBP4 and SPP1 polymorphisms on age at loss of ambulation (LoA) in a multiethnic Duchenne muscular dystrophy (DMD) cohort. Methods We genotyped SPP1 rs28357094 and LTBP4 haplotype in 283 of 340 participants in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). Median ages at LoA were compared by Kaplan-Meier analysis and log-rank test. We controlled polymorphism analyses for concurrent effects of glucocorticoid corticosteroid (GC) treatment (time-varying Cox regression) and for population stratification (multidimensional scaling of genome-wide markers). Results Hispanic and South Asian participants (n = 18, 41) lost ambulation 2.7 and 2 years earlier than Caucasian subjects ( p = 0.003, <0.001). The TG/GG genotype at SPP1 rs28357094 was associated to 1.2-year-earlier median LoA ( p = 0.048). This difference was greater (1.9 years, p = 0.038) in GC-treated participants, whereas no difference was observed in untreated subjects. Cox regression confirmed a significant effect of SPP1 genotype in GC-treated participants (hazard ratio = 1.61, p = 0.016). LTBP4 genotype showed a direction of association with age at LoA as previously reported, but it was not statistically significant. After controlling for population stratification, we confirmed a strong effect of LTBP4 genotype in Caucasians (2.4 years, p = 0.024). Median age at LoA with the protective LTBP4 genotype in this cohort was 15.0 years, 16.0 for those who were treated with GC. Interpretation SPP1 rs28357094 acts as a pharmacodynamic biomarker of GC response, and LTBP4 haplotype modifies age at LoA in the CINRG-DNHS cohort. Adjustment for GC treatment and population stratification appears crucial in assessing genetic modifiers in DMD. Ann Neurol 2015;77:684-696 [ABSTRACT FROM AUTHOR]

Published
2015
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218. Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy.

Author

Connolly, Anne M., Malkus, Elizabeth C., Mendell, Jerry R., Flanigan, Kevin M., Miller, J. Philip, Schierbecker, Jeanine R., Siener, Catherine A., Golumbek, Paul T., Zaidman, Craig M., Mcdonald, Craig M., Johnson, Linda, Nicorici, Alina, Karachunski, Peter I., Day, John W., Kelecic, Jason M., Lowes, Linda P., Alfano, Lindsay N., Darras, Basil T., Kang, Peter B., and Quigley, Janet

Abstract

ABSTRACT Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD ( N = 91; 16.7 ± 4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8 ± 22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use. Muscle Nerve 51: 522-532, 2015 [ABSTRACT FROM AUTHOR]

Published
2015
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225. Chapter 7: Electrodiagnosis in Pediatrics.

Author

McDonald, Craig M.

Subjects
*ELECTRODIAGNOSIS, *ELECTRICITY in medicine, *CARE of children with disabilities
Abstract

Chapter 7 of the book "Pediatric Rehabilitation: Principles and Practice," Fourth Edition, edited by Michael A. Alexander and Dennis J. Matthews is presented. It explores the aspects to consider in pediatric electrodiagnosis. It discusses the technical factors associated with each type of electrodiagnosis. It tackles the clinical problems associated with such diagnosis of children with disabilities.

Published
2009

226. Chapter 12: Neuromuscular Diseases.

Author

McDonald, Craig M.

Subjects
*NEUROMUSCULAR diseases in children, *REHABILITATION of children with disabilities, *EXERCISE
Abstract

Chapter 12 of the book "Pediatric Rehabilitation: Principles and Practice," Fourth Edition, edited by Michael A. Alexander and Dennis J. Matthews is presented. It explores the diagnostic evaluation and natural history of neuromuscular diseases. It discusses the rehabilitation of children with such disease. It tackles the strengthening exercise for childhood neuromuscular disease.

Published
2009

237. Duchenne Muscular Dystrophy

Author

McDonald, Craig M., primary, Abresch, Richard T., additional, Carter, Gregory T., additional, Fowler, William M., additional, Johnson, E Ralph, additional, Kilmer, David D., additional, and Sigford, Barbara J., additional

Published
1995
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238. Limb-Girdle Syndromes

Author

McDonald, Craig M., primary, Johnson, E Ralph, additional, Abresch, Richard T., additional, Carter, Gregory T., additional, Fowler, William M., additional, and Kilmer, David D., additional

Published
1995
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240. Spinal Muscular Atrophy

Author

Carter, Gregory T., primary, Abresch, Richard T., additional, Fowler, William M., additional, Johnson, E Ralph, additional, Kilmer, David D., additional, and McDonald, Craig M., additional

Published
1995
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241. Becker??s Muscular Dystrophy

Author

McDonald, Craig M., primary, Abresch, Richard T., additional, Carter, Gregory T., additional, Fowler, William M., additional, Johnson, E Ralph, additional, and Kilmer, David D., additional

Published
1995
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242. Facioscapulohumeral Muscular Dystrophy

Author

Kilmer, David D., primary, Abresch, Richard T., additional, McCrory, Megan A., additional, Carter, Gregory T., additional, Fowler, William M., additional, Johnson, E Ralph, additional, and McDonald, Craig M., additional

Published
1995
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243. Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies

Author

Spurney, Christopher F., McCaffrey, Francis M., Cnaan, Avital, Morgenroth, Lauren P., Ghelani, Sunil J., Gordish-Dressman, Heather, Arrieta, Adrienne, Connolly, Anne M., Lotze, Timothy E., McDonald, Craig M., Leshner, Robert T., and Clemens, Paula R.

Abstract

Cardiac disease is a major cause of death in patients with muscular dystrophies. The use of feasible and reproducible echocardiographic measures of cardiac function is critical to advance the field of therapeutics for dystrophic cardiomyopathy.

Published
2015
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244. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

Author

Buyse, Gunnar M, Voit, Thomas, Schara, Ulrike, Straathof, Chiara S M, D'Angelo, M Grazia, Bernert, Günther, Cuisset, Jean-Marie, Finkel, Richard S, Goemans, Nathalie, McDonald, Craig M, Rummey, Christian, and Meier, Thomas

Abstract

Cardiorespiratory failure is the leading cause of death in Duchenne muscular dystrophy. Based on preclinical and phase 2 evidence, we assessed the efficacy and safety of idebenone in young patients with Duchenne muscular dystrophy who were not taking concomitant glucocorticoids.

Published
2015
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245. Evaluation of Phrenic Nerve and Diaphragm Function with Peripheral Nerve Stimulation and M-Mode Ultrasonography in Potential Pediatric Phrenic Nerve or Diaphragm Pacing Candidates

Author

Skalsky, Andrew J., Lesser, Daniel J., and McDonald, Craig M.

Abstract

Assessing phrenic nerve function in the setting of diaphragmatic paralysis in diaphragm pacing candidates can be challenging. Traditional imaging modalities and electrodiagnostic evaluations are technically difficult. Either modality alone is not a direct measure of the function of the phrenic nerve and diaphragm unit. In this article, the authors present their method for evaluating phrenic nerve function and the resulting diaphragm function. Stimulating the phrenic nerve with transcutaneous stimulation and directly observing the resulting movement of the hemidiaphragm with M-mode ultrasonography provides quantitative data for predicting the success of advancing technologies such as phrenic nerve pacing and diaphragm pacing.

Published
2015
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249. Letters to the editor

Author

Eraser, Jeffrey L., primary, Bromberg, Mark B., additional, Albers, James W., additional, Robinson, Lawrence R., additional, Henderson, Marjorie, additional, Sheean, Geoffrey, additional, McDonald, Craig M., additional, Bötzel, Kai, additional, Witt, Thomas N., additional, Herskovitz, Steven, additional, Bieri, Phyllis L., additional, Berger, Alan R., additional, Tanaka, Masami, additional, Yoshimoto, Haruko, additional, Safto, Yutaka, additional, Yamazaki, Motoyoshi, additional, Tsuji, Shoji, additional, van der Kamp, W., additional, Zwinderman, A. H., additional, Roos, R. A. C., additional, Ferrari, M. D., additional, Kamphuisen, H. A. C., additional, van Dijk, J. G., additional, Morgenlander, Joel C., additional, Leis, A. Arturo, additional, Schild, John H., additional, and Stokić, Dobrivoje S., additional

Published
1994
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250. THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study.

Author

Mcdonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine M., Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, and Peltz, Stuart

Abstract

ABSTRACT Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double-blind, placebo-controlled trial of ataluren. Placebo arm data ( N = 57) provided insight into the natural history of the 6-minute walk test (6MWT) and other endpoints. Methods: Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry. Results: Baseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. Conclusion: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials. Muscle Nerve 48: 343-356, 2013 [ABSTRACT FROM AUTHOR]

Published
2013
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