Factors Associated With Health-Related Quality of Life in Children With Duchenne Muscular Dystrophy

Wei, Yuanyuan; Speechley, Kathy N.; Zou, Guangyong; Campbell, Craig

doi:10.1177/0883073815627879

Cited by 34 publications

(42 citation statements)

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“…Additional musculoskeletal, respiratory, and cardiac complications emerge as DMD advances. 3 Subjective fatigue, the focus of this study, is defined as a lack of energy or the existence of weakness or exhaustionmentally, physically, or both. 2 Improved management with corticosteroid therapy and non-invasive ventilation (NIV) has prolonged survival.…”

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confidence: 99%

Fatigue in young people with Duchenne muscular dystrophy

El-Aloul

Speechley

Wei

et al. 2019

Develop Med Child Neuro

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AIM To describe fatigue in Duchenne muscular dystrophy (DMD) from patients' and parents' perspectives and to explore risk factors for fatigue in children and adolescents with DMD.METHOD A multicentre, cross-sectional study design was used. Seventy-one patients (all males; median age 12y, age range 5-17y) identified via the Canadian Neuromuscular Disease Registry, and their parents completed questionnaires. Subjective fatigue was assessed using the Pediatric Quality of Life Inventory Multidimensional Fatigue Scale by child self-report and parent proxy-report. RESULTSPatients with DMD across ages and disease stages experienced greater fatigue compared to typically developing controls from published data. Sleep disturbance symptoms were associated with greater fatigue by child self-report (q=-0.42; p=0.003) and parent proxyreport (q=-0.51; p<0.001). Depressive symptoms were associated with greater fatigue by child self-report (q=-0.46; p<0.001) and parent proxy-report (q=-0.45; p<0.001). Lower functional ability was associated with greater fatigue by parent proxy-report (q=0.26; p=0.03). Physical activity level, and musculoskeletal, respiratory, and cardiac function were not associated with fatigue.

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confidence: 99%

Fatigue in young people with Duchenne muscular dystrophy

El-Aloul

Speechley

Wei

et al. 2019

Develop Med Child Neuro

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“…(3) execution of different tests with different input data, especially on the BGQ; (4) analysis and selection of the obtained results in the collaborative work, to decide which of the new protein sequences would be produced as actual synthetic proteins for wet-lab experiments. Neither enhancements nor additional code development were designed or implemented to perform the experiments with InSiPS.…”

Section: Scope Of the Thesismentioning

confidence: 99%

“…The parameter values that were used as input for the different experiments are listed in Table 6. 4.…”

Section: Genetic Algorithm Parametersmentioning

confidence: 99%

“…6. 4 The maximum execution time for each job submitted is 24 hours and there is no limit on the number of jobs. However, the start time for a submitted job depends on, among other variables, the number of nodes requested, the maximum execution time limit (wall clock limit) and the current availability of resources (i.e., how many jobs are in the queue).…”

Section: Carleton Bioinformatics Data Lab Clustermentioning

confidence: 99%

“…The Canadian Neuromuscular Disease Registry (CNDR) collected information on patients with neuromuscular diseases across Canada [3], among which 253 with muscular dystrophy were included. Via the CNDR registry, a study based on surveys to families of 176 children -between 4 and 18 years of age -with DMD reported that fatigue and the need for wheelchair use severely impacted health-related quality of life in patients [4]. One attempt to combat this degenerative disease involves a joint effort along two independent research lines, with investigators drawn from the disciplines of data science and biochemistry.…”

Section: Introduction 11 Overview Of the Thesismentioning

confidence: 99%

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Data Science Research to Support Stem Cell Therapy for Muscular Dystrophy

Salas¹,

Nathaly²

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This thesis project focused on using a sequence-based, high-performance computational tool to design synthetic proteins and is part of current collaborative research on Duchenne Muscular Dystrophy (DMD). A possible treatment for DMD consists of injecting patients with healthy muscle satellite cells grown in tissue culture. However, such cells cannot currently be produced in quantity because they convert to muscle cells (differentiate) prematurely. Using InSiPS, the In-Silico Protein Synthesizer, protein sequences were designed to interact with target proteins and inhibit the protein-protein interaction proposed to regulate the premature differentiation. The resulting sequences were predicted to interact with the target proteins with high specificity (99.98%). Complementary biochemistry experiments indicated interactions with the intended target for two out of ten synthetic proteins. These results are being studied as part of the ongoing research seeking to develop a treatment for DMD. I would like to acknowledge and give my sincerest appreciation to the person who made possible my participation in this research project, Dr. Frank Dehne. I will always be grateful for having such a recognized investigator as my thesis supervisor, who was always happy to help and give advice with his contagious passion for research that motivated me to improve myself. An enormous thank you to Dr. Andrew Schoenrock, for his infinite patience in helping me at every step of the project, and whose expertise and knowledge of PIPE and InSiPS were crucial for my progress. Also, a big thank to Daniel Burnside for kindly helping me to understand and to explain biochemistry concepts. I also thank Dr. Ashkan Golshani and everyone in the Carleton Bioinformatics group. I was very lucky to be among dedicated investigators who were always happy to provide ideas to improve the present work. Special thanks go to Dr. Alexandre Blais, who developed the initial research proposal, who designed the biochemical experimental validation and who led the group that performed it-all with great commitment and a high sense of collaboration. My infinite appreciation goes also to Carole Love, and all the hard-work she put into proofreading and helping me with suggestions that make me feel proud of my thesis writing. I want to express my deepest gratitude to the Mexican National Council for Science and Technology (CONACyT) for sponsoring my graduate studies on behalf of my country-México-and for helping me to pursue this major milestone in my life. I am very fortunate to have in my life a special colleague who happens to be my husband and love of my life. Thank you, Pablo for your unending support and for sharing with me this adventure.

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Use of a wearable device to assess sleep and motor function in Duchenne muscular dystrophy

et al. 2019

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Introduction: Rest-activity disruption is an important feature of Duchenne muscular dystrophy (DMD). We sought to describe sleep impairment and its relationship to quality of life (QOL) and to evaluate associations between rest-activity, sleep quality, and 6-minute walk test (6MWT) in DMD.Methods: Sleep impairment and its relationship to QOL was assessed by questionnaire in 54 children (33 ambulatory, 21 nonambulatory) with DMD. Rest-activity characteristics were calculated for 23 of these children (14 ambulatory, nine nonambulatory) by actigraphy.Results: Pathologic sleep was reported in 11 (20%) participants and correlated with lower QOL but not with ambulatory status. In ambulatory participants who completed actigraphy, rest-activity rhythm fragmentation was associated with subjective sleep impairment. Habitual daytime activity level was associated with 6MWT performance.Discussion: Children with DMD experience substantial sleep impairment that is related to QOL. Wrist actigraphy may be a parsimonious tool for monitoring both sleep and motor impairment in ambulatory children with DMD.

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Factors Associated With Health-Related Quality of Life in Children With Duchenne Muscular Dystrophy

Cited by 34 publications

References 40 publications

Fatigue in young people with Duchenne muscular dystrophy

Fatigue in young people with Duchenne muscular dystrophy

Data Science Research to Support Stem Cell Therapy for Muscular Dystrophy

Use of a wearable device to assess sleep and motor function in Duchenne muscular dystrophy

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