Search

Your search keyword '"Willcocks, Rebecca J"' showing total 159 results
Start Over Author "Willcocks, Rebecca J"
159 results on '"Willcocks, Rebecca J"'

2. Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging.

Author

Batra, Abhinandan, Barnard, Alison M, Lott, Donovan J, Willcocks, Rebecca J, Forbes, Sean C, Chakraborty, Saptarshi, Daniels, Michael J, Arbogast, Jannik, Triplett, William, Henricson, Erik K, Dayan, Jonathan G, Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J, McDonald, Craig M, Vandenborne, Krista, and Walter, Glenn A

Subjects
Humans, Muscular Dystrophy, Duchenne, Cardiomyopathies, Magnetic Resonance Imaging, Magnetic Resonance Imaging, Cine, Stroke Volume, Prospective Studies, Ventricular Function, Left, Adolescent, Child, Child, Preschool, Cardiac circumferential strain, Cardiac magnetic resonance imaging, Duchenne muscular dystrophy, Heart Disease, Brain Disorders, Pediatric, Muscular Dystrophy, Intellectual and Developmental Disabilities (IDD), Clinical Research, Cardiovascular, Rare Diseases, Biomedical Imaging, Duchenne/ Becker Muscular Dystrophy, Cardiorespiratory Medicine and Haematology, Cardiovascular System & Hematology
Abstract

BackgroundThe lack of dystrophin in cardiomyocytes in Duchenne muscular dystrophy (DMD) is associated with progressive decline in cardiac function eventually leading to death by 20-40 years of age. The aim of this prospective study was to determine rate of progressive decline in left ventricular (LV) function in Duchenne muscular dystrophy (DMD) over 5 years.MethodsShort axis cine and grid tagged images of the LV were acquired in individuals with DMD (n = 59; age = 5.3-18.0 years) yearly, and healthy controls at baseline (n = 16, age = 6.0-18.3 years) on a 3 T MRI scanner. Grid-tagged images were analyzed for composite circumferential strain (ℇcc%) and ℇcc% in six mid LV segments. Cine images were analyzed for left ventricular ejection fraction (LVEF), LV mass (LVM), end-diastolic volume (EDV), end-systolic volume (ESV), LV atrioventricular plane displacement (LVAPD), and circumferential uniformity ratio estimate (CURE). LVM, EDV, and ESV were normalized to body surface area for a normalized index of LVM (LVMI), EDV (EDVI) and ESV (ESVI).ResultsAt baseline, LV ℇcc% was significantly worse in DMD compared to controls and five of the six mid LV segments demonstrated abnormal strain in DMD. Longitudinal measurements revealed that ℇcc% consistently declined in individuals with DMD with the inferior segments being more affected. LVEF progressively declined between 3 to 5 years post baseline visit. In a multivariate analysis, the use of cardioprotective drugs trended towards positively impacting cardiac measures while loss of ambulation and baseline age were associated with negative impact. Eight out of 17 cardiac parameters reached a minimal clinically important difference with a threshold of 1/3 standard deviation.ConclusionThe study shows a worsening of circumferential strain in dystrophic myocardium. The findings emphasize the significance of early and longitudinal assessment of cardiac function in DMD and identify early biomarkers of cardiac dysfunction to help design clinical trials to mitigate cardiac pathology. This study provides valuable non-invasive and non-contrast based natural history data of cardiac changes which can be used to design clinical trials or interpret the results of current trials aimed at mitigating the effects of decreased cardiac function in DMD.

Published
2022

5. Magnetic Resonance Imaging Studies in Duchenne Muscular Dystrophy: Linking Findings to the Physical Therapy Clinic

Author

Senesac, Claudia R., Barnard, Alison M., Lott, Donovan J., Nair, Kavya S., Harrington, Ann T., Willcocks, Rebecca J., Zilke, Kirsten L., Rooney, William D., Walter, Glenn A., and Vandenborne, Krista

Subjects
Diagnosis, Care and treatment, Usage, Research, Physical therapy -- Research, Duchenne muscular dystrophy -- Diagnosis -- Care and treatment, Medical research, Magnetic resonance imaging -- Usage, Medicine, Experimental, Therapeutics, Physiological -- Research
Abstract

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder resulting in skeletal muscle weakness, cardiac impairment, and respiratory insufficiency. (1-3) DMD is caused by mutations in the X chromosome [...], Duchenne muscular dystrophy (DMD) is a muscle degenerative disorder that manifests in early childhood and results in progressive muscle weakness. Physical therapists have long been an important component of the multidisciplinary team caring for people with DMD, providing expertise in areas of disease assessment, contracture management, assistive device prescription, and exercise prescription. Over the last decade, magnetic resonance imaging of muscles in people with DMD has led to an improved understanding of the muscle pathology underlying the clinical manifestations of DMD. Findings from magnetic resonance imaging (MRI) studies in DMD, paired with the clinical expertise of physical therapists, can help guide research that leads to improved physical therapist care for this unique patient population. The 2 main goals of this perspective article are to (1) summarize muscle pathology and disease progression findings from qualitative and quantitative muscle MRI studies in DMD and (2) link MRI findings of muscle pathology to the clinical manifestations observed by physical therapists with discussion of any potential implications of MRI findings on physical therapy management.

Published
2020
Full Text
View/download PDF

7. Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy

Author

Willcocks, Rebecca J., primary, Barnard, Alison M., additional, Daniels, Michael J., additional, Forbes, Sean C., additional, Triplett, William T., additional, Brandsema, John F., additional, Finanger, Erika L., additional, Rooney, William D., additional, Kim, Sarah, additional, Wang, Dah‐Jyuu, additional, Lott, Donovan J., additional, Senesac, Claudia R., additional, Walter, Glenn A., additional, Sweeney, H. Lee, additional, and Vandenborne, Krista, additional

Published
2023
Full Text
View/download PDF

8. Modeling disease trajectory in Duchenne muscular dystrophy

Author

Rooney, William D., Berlow, Yosef A., Triplett, William T., Forbes, Sean C., Willcocks, Rebecca J., Wang, Dah-Jyuu, Arpan, Ishu, Arora, Harneet, Senesac, Claudia, Lott, Donovan J., Tennekoon, Gihan, Finkel, Richard, Russman, Barry S., Finanger, Erika L., Chakraborty, Saptarshi, OʼBrien, Elliott, Moloney, Brendan, Barnard, Alison, Sweeney, H. Lee, Daniels, Michael J., Walter, Glenn A., and Vandenborne, Krista

Published
2020
Full Text
View/download PDF

9. MR biomarkers predict clinical function in Duchenne muscular dystrophy

Author

Barnard, Alison M., Willcocks, Rebecca J., Triplett, William T., Forbes, Sean C., Daniels, Michael J., Chakraborty, Saptarshi, Lott, Donovan J., Senesac, Claudia R., Finanger, Erika L., Harrington, Ann T., Tennekoon, Gihan, Arora, Harneet, Wang, Dah-Jyuu, Sweeney, H. Lee, Rooney, William D., Walter, Glenn A., and Vandenborne, Krista

Published
2020
Full Text
View/download PDF

10. Multivariate modeling of magnetic resonance biomarkers and clinical outcome measures for Duchenne muscular dystrophy clinical trials

Author

Kim, Sarah, primary, Willcocks, Rebecca J., additional, Daniels, Michael J., additional, Morales, Juan Francisco, additional, Yoon, Deok Yong, additional, Triplett, William T., additional, Barnard, Alison M., additional, Conrado, Daniela J., additional, Aggarwal, Varun, additional, Belfiore‐Oshan, Ramona, additional, Martinez, Terina N., additional, Walter, Glenn A., additional, Rooney, William D., additional, and Vandenborne, Krista, additional

Published
2023
Full Text
View/download PDF

11. Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy.

Author

Willcocks, Rebecca J., Barnard, Alison M., Daniels, Michael J., Forbes, Sean C., Triplett, William T., Brandsema, John F., Finanger, Erika L., Rooney, William D., Kim, Sarah, Wang, Dah‐Jyuu, Lott, Donovan J., Senesac, Claudia R., Walter, Glenn A., Sweeney, H. Lee, and Vandenborne, Krista

Subjects
*DUCHENNE muscular dystrophy, *MAGNETIC resonance, *PAIN threshold, *MUSCULAR dystrophy, *LEG muscles, *SPECTRAL imaging
Abstract

Objective: Magnetic resonance (MR) measures of muscle quality are highly sensitive to disease progression and predictive of meaningful functional milestones in Duchenne muscular dystrophy (DMD). This investigation aimed to establish the reproducibility, responsiveness to disease progression, and minimum clinically important difference (MCID) for multiple MR biomarkers at different disease stages in DMD using a large natural history dataset. Methods: Longitudinal MR imaging and spectroscopy outcomes and ambulatory function were measured in 180 individuals with DMD at three sites, including repeated measurements on two separate days (within 1 week) in 111 participants. These data were used to calculate day‐to‐day reproducibility, responsiveness (standardized response mean, SRM), minimum detectable change, and MCID. A survey of experts was also performed. Results: MR spectroscopy fat fraction (FF), as well as MR imaging transverse relaxation time (MRI‐T2), measures performed in multiple leg muscles, and had high reproducibility (Pearson's R > 0.95). Responsiveness to disease progression varied by disease stage across muscles. The average FF from upper and lower leg muscles was highly responsive (SRM > 0.9) in both ambulatory and nonambulatory individuals. MCID estimated from the distribution of scores, by anchoring to function, and via expert opinion was between 0.01 and 0.05 for FF and between 0.8 and 3.7 ms for MRI‐T2. Interpretation: MR measures of FF and MRI T2 are reliable and highly responsive to disease progression. The MCID for MR measures is less than or equal to the typical annualized change. These results confirm the suitability of these measures for use in DMD and potentially other muscular dystrophies. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

12. Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

Author

Comi, Giacomo P., primary, Niks, Erik H., additional, Vandenborne, Krista, additional, Cinnante, Claudia M., additional, Kan, Hermien E., additional, Willcocks, Rebecca J., additional, Velardo, Daniele, additional, Magri, Francesca, additional, Ripolone, Michela, additional, van Benthem, Jules J., additional, van de Velde, Nienke M., additional, Nava, Simone, additional, Ambrosoli, Laura, additional, Cazzaniga, Sara, additional, and Bettica, Paolo U., additional

Published
2023
Full Text
View/download PDF

14. Step Activity Monitoring in Boys with Duchenne Muscular Dystrophy and its Correlation with Magnetic Resonance Measures and Functional Performance

Author

Nair, Kavya S., primary, Lott, Donovan J., additional, Forbes, Sean C., additional, Barnard, Alison M., additional, Willcocks, Rebecca J., additional, Senesac, Claudia R., additional, Daniels, Michael J., additional, Harrington, Ann T., additional, Tennekoon, Gihan I., additional, Zilke, Kirsten, additional, Finanger, Erika L., additional, Finkel, Richard S., additional, Rooney, William D., additional, Walter, Glenn A., additional, and Vandenborne, Krista, additional

Published
2022
Full Text
View/download PDF

15. Development of Contractures in DMD in Relation to MRI-Determined Muscle Quality and Ambulatory Function

Author

Willcocks, Rebecca J., primary, Barnard, Alison M., additional, Wortman, Ryan J., additional, Senesac, Claudia R., additional, Lott, Donovan J., additional, Harrington, Ann T., additional, Zilke, Kirsten L., additional, Forbes, Sean C., additional, Rooney, William D., additional, Wang, Dah-Jyuu, additional, Finanger, Erika L., additional, Tennekoon, Gihan I., additional, Daniels, Michael J., additional, Triplett, William T., additional, Walter, Glenn A., additional, and Vandenborne, Krista, additional

Published
2022
Full Text
View/download PDF

16. Additional file 3 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities, sense organs, skin and connective tissue diseases, nervous system diseases
Abstract

Additional file 3: Longitudinal change in global strain for mid ventricle of DMD.

Published
2022
Full Text
View/download PDF

17. Additional file 4 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Abstract

Additional file 4: Peak and global mid ventricular strain (εcc %) in unaffected controls (n=15) and individuals with DMD (n=46) at baseline (UF Cohort).

Published
2022
Full Text
View/download PDF

18. Additional file 2 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Abstract

Additional file 2: Longitudinal changes in global strain in DMD. Solid line for global strain was defined based on normal zone cut off of -17% as given by HARP software. Red lines indicates subjects with more than 5 years data, filled triangles represent unaffected controls.

Published
2022
Full Text
View/download PDF

19. Additional file 6 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 6: Comparison of cardiac function in control and DMD subjects at baseline (UF Cohort).

Published
2022
Full Text
View/download PDF

20. Additional file 5 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 5: Strain for each LV segment in controls and individuals with DMD (n=46) at baseline (UF cohort).

Published
2022
Full Text
View/download PDF

21. Additional file 1 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 1: Global mid ventricular strain (εcc %) in unaffected controls (n=15) and individuals with DMD (n=58) at baseline.

Published
2022
Full Text
View/download PDF

22. Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort

Author

Willcocks, Rebecca J., Rooney, William D., Triplett, William T., Forbes, Sean C., Lott, Donovan J., Senesac, Claudia R., Daniels, Michael J., Wang, Dah-Jyuu, Harrington, Ann T., Tennekoon, Gihan I., Russman, Barry S., Finanger, Erika L., Byrne, Barry J., Finkel, Richard S., Walter, Glenn A., Sweeney, Lee H., and Vandenborne, Krista

Published
2016
Full Text
View/download PDF

23. Characterization of patients with Becker muscular dystrophy by histology, magnetic resonance imaging, function, and strength assessments

Author

Comi, Giacomo P., primary, Niks, Erik H., additional, Cinnante, Claudia M., additional, Kan, Hermien E., additional, Vandenborne, Krista, additional, Willcocks, Rebecca J., additional, Velardo, Daniele, additional, Ripolone, Michela, additional, van Benthem, Jules J., additional, van de Velde, Nienke M., additional, Nava, Simone, additional, Ambrosoli, Laura, additional, Cazzaniga, Sara, additional, and Bettica, Paolo U., additional

Published
2021
Full Text
View/download PDF

26. Walking activity in a large cohort of boys with Duchenne muscular dystrophy

Author

Lott, Donovan J., primary, Taivassalo, Tanja, additional, Senesac, Claudia R., additional, Willcocks, Rebecca J., additional, Harrington, Ann M., additional, Zilke, Kirsten, additional, Cunkle, Hilary, additional, Powers, Catherine, additional, Finanger, Erika L., additional, Rooney, William D., additional, Tennekoon, Gihan I., additional, and Vandenborne, Krista, additional

Published
2020
Full Text
View/download PDF

27. Upper and Lower Extremities in Duchenne Muscular Dystrophy Evaluated with Quantitative MRI and Proton MR Spectroscopy in a Multicenter Cohort

Author

Forbes, Sean C., primary, Arora, Harneet, additional, Willcocks, Rebecca J., additional, Triplett, William T., additional, Rooney, William D., additional, Barnard, Alison M., additional, Alabasi, Umar, additional, Wang, Dah-Jyuu, additional, Lott, Donovan J., additional, Senesac, Claudia R., additional, Harrington, Ann T., additional, Finanger, Erika L., additional, Tennekoon, Gihan I., additional, Brandsema, John, additional, Daniels, Michael J., additional, Sweeney, H. Lee, additional, Walter, Glenn A., additional, and Vandenborne, Krista, additional

Published
2020
Full Text
View/download PDF

29. Conference report on contractures in musculoskeletal and neurological conditions

Author

Nuckolls, Glen H., primary, Kinnett, Kathi, additional, Dayanidhi, Sudarshan, additional, Domenighetti, Andrea A., additional, Duong, Tina, additional, Hathout, Yetrib, additional, Lawlor, Michael W., additional, Lee, Sabrina S. M., additional, Magnusson, S. Peter, additional, McDonald, Craig M., additional, McNally, Elizabeth M., additional, Miller, Natalie F., additional, Olwin, Bradley B., additional, Raghavan, Preeti, additional, Roberts, Thomas J., additional, Rutkove, Seward B., additional, Sarwark, John F., additional, Senesac, Claudia R., additional, Vogel, Leslie F., additional, Walter, Glenn A., additional, Willcocks, Rebecca J., additional, Rymer, William Z., additional, and Lieber, Richard L., additional

Published
2020
Full Text
View/download PDF

31. Characterization of patients with Becker muscular dystrophy by histology, magnetic resonance imaging, function, and strength assessments.

Author

Comi, Giacomo P., Niks, Erik H., Cinnante, Claudia M., Kan, Hermien E., Vandenborne, Krista, Willcocks, Rebecca J., Velardo, Daniele, Ripolone, Michela, van Benthem, Jules J., van de Velde, Nienke M., Nava, Simone, Ambrosoli, Laura, Cazzaniga, Sara, and Bettica, Paolo U.

Abstract

Introduction/Aims: Becker muscular dystrophy (BMD) is characterized by variable disease severity and progression, prompting the identification of biomarkers for clinical trials. We used data from an ongoing phase II study to provide a comprehensive characterization of a cohort of patients with BMD, and to assess correlations between histological and magnetic resonance imaging (MRI) markers with muscle function and strength. Methods: Eligible patients were ambulatory males with BMD, aged 18 to 65 years (200 to 450 meters on 6‐minute walk test). The following data were obtained: function test results, strength, fat‐fraction quantification using chemical shift‐encoded MRI (whole thigh and quadriceps), and fibrosis and muscle fiber area (MFA) of the brachial biceps. Results: Of 70 patients screened, 51 entered the study. There was substantial heterogeneity between patients in muscle morphology (histology and MRI), with high fat replacement. Total fibrosis correlated significantly and mostly moderately with all functional endpoints, including both upper arm strength assessments (left and right elbow flexion rho −.574 and −.588, respectively [both P <.0001]), as did MRI fat fraction (whole thigh and quadriceps), for example, with four‐stair‐climb velocity −.554 and −.550, respectively (both P <.0001). Total fibrosis correlated significantly and moderately with both MRI fat fraction assessments (.500 [P =.0003] and.423 [.0024], respectively). Discussion: In this BMD cohort, micro‐ and macroscopic morphological muscle parameters correlated moderately with each other and with functional parameters, potentially supporting the use of MRI fat fraction and histology as surrogate outcome measures in patients with BMD, although additional research is required to validate this. [ABSTRACT FROM AUTHOR]

Published
2022
Full Text
View/download PDF

34. Conference report on contractures in musculoskeletal and neurological conditions

Author

Nuckolls, Glen H., Kinnett, Kathi, Dayanidhi, Sudarshan, Domenighetti, Andrea A., Duong, Tina, Hathout, Yetrib, Lawlor, Michael W., Lee, Sabrina S.M., Magnusson, S. Peter, McDonald, Craig M., McNally, Elizabeth M., Miller, Natalie F., Olwin, Bradley B., Raghavan, Preeti, Roberts, Thomas J., Rutkove, Seward B., Sarwark, John F., Senesac, Claudia R., Vogel, Leslie F., Walter, Glenn A., Willcocks, Rebecca J., Rymer, William Z., Lieber, Richard L., Nuckolls, Glen H., Kinnett, Kathi, Dayanidhi, Sudarshan, Domenighetti, Andrea A., Duong, Tina, Hathout, Yetrib, Lawlor, Michael W., Lee, Sabrina S.M., Magnusson, S. Peter, McDonald, Craig M., McNally, Elizabeth M., Miller, Natalie F., Olwin, Bradley B., Raghavan, Preeti, Roberts, Thomas J., Rutkove, Seward B., Sarwark, John F., Senesac, Claudia R., Vogel, Leslie F., Walter, Glenn A., Willcocks, Rebecca J., Rymer, William Z., and Lieber, Richard L.

Abstract

Limb contractures are debilitating complications associated with various muscle and nervous system disorders. This report summarizes presentations at a conference at the Shirley Ryan AbilityLab in Chicago, Illinois, on April 19–20, 2018, involving researchers and physicians from diverse disciplines who convened to discuss current clinical and preclinical understanding of contractures in Duchenne muscular dystrophy, stroke, cerebral palsy, and other conditions. Presenters described changes in muscle architecture, activation, extracellular matrix, satellite cells, and muscle fiber sarcomeric structure that accompany or predispose muscles to contracture. Participants identified ongoing and future research directions that may lead to understanding of the intersecting factors that trigger contractures. These include additional studies of changes in muscle, tendon, joint, and neuronal tissues during contracture development with imaging, molecular, and physiologic approaches. Participants identified the requirement for improved biomarkers and outcome measures to identify patients likely to develop contractures and to accurately measure efficacy of treatments currently available and under development.

Published
2020

35. Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history

Author

Arora, Harneet, Willcocks, Rebecca J, Lott, Donovan J, Harrington, Ann T, Senesac, Claudia R, Zilke, Kirsten L, Daniels, Michael J, Xu, Dandan, Tennekoon, Gihan I, Finanger, Erika L, Russman, Barry S, Finkel, Richard S, Triplett, William T, Byrne, Barry J, Walter, Glenn A, Sweeney, H Lee, and Vandenborne, Krista

Subjects
Male, Time Factors, Adolescent, Age Factors, Walk Test, Walking, Magnetic Resonance Imaging, Article, Cohort Studies, Muscular Dystrophy, Duchenne, Child, Preschool, Outcome Assessment, Health Care, Exercise Test, Disease Progression, Image Processing, Computer-Assisted, Humans, Female, Child
Abstract

Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5-12.9-year-olds.Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6-minute walk test (6MWT) and timed function tests (TFT: 10-m walk/run, climbing 4 stairs, supine to stand).Boys with DMD had impaired functional performance even at 5-6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7-12.9 year olds.This study provides insight into the contemporary natural history of key functional endpoints in DMD. Muscle Nerve 58: 631-638, 2018.

Published
2017

37. Walking activity in a large cohort of boys with Duchenne muscular dystrophy.

Author

Lott, Donovan J., Taivassalo, Tanja, Senesac, Claudia R., Willcocks, Rebecca J., Harrington, Ann M., Zilke, Kirsten, Cunkle, Hilary, Powers, Catherine, Finanger, Erika L., Rooney, William D., Tennekoon, Gihan I., and Vandenborne, Krista

Abstract

Introduction: In this study we explored walking activity in a large cohort of boys with Duchenne muscular dystrophy (DMD). Methods: Step activity (monitored for 7 days), functional ability, and strength were quantified in ambulatory boys (5‐12.9 years of age) with DMD and unaffected boys. Ambulatory status was determined 2 years later. Results: Two to 5 days of activity monitoring predicted weekly step activity (adjusted R2 = 0.80‐0.95). Age comparisons revealed significant declines for step activity with increasing age, and relationships were found between step activity with both function and strength (P <.01). Our regression model predicted 36.5% of the variance in step activity. Those who were still ambulatory after 2 years demonstrated baseline step activity nearly double that of those who were no longer walking 2 years later (P <.01). Discussion: Step activity for DMD is related to and predictive of functional declines, which may be useful for clinical trials. [ABSTRACT FROM AUTHOR]

Published
2021
Full Text
View/download PDF

38. Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history

Author

Arora, Harneet, primary, Willcocks, Rebecca J., additional, Lott, Donovan J., additional, Harrington, Ann T., additional, Senesac, Claudia R., additional, Zilke, Kirsten L., additional, Daniels, Michael J., additional, Xu, Dandan, additional, Tennekoon, Gihan I., additional, Finanger, Erika L., additional, Russman, Barry S., additional, Finkel, Richard S., additional, Triplett, William T., additional, Byrne, Barry J., additional, Walter, Glenn A., additional, Sweeney, H. Lee, additional, and Vandenborne, Krista, additional

Published
2018
Full Text
View/download PDF

39. MoveDMD®: Positive Effects of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension (S29.006)

Author

Finkel, Richard, primary, Vandenborne, Krista H. E., additional, Sweeney, H. Lee, additional, Finanger, Erika, additional, Tennekoon, Gihan, additional, Shieh, Perry, additional, Willcocks, Rebecca J., additional, Forbes, Sean C., additional, Triplett, William, additional, Yum, Sabrina, additional, Mancini, Maria, additional, Fretzen, Angelika, additional, and Donovan, Joanne, additional

Published
2018
Full Text
View/download PDF

40. Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy

Author

Barnard, Alison M., primary, Willcocks, Rebecca J., additional, Finanger, Erika L., additional, Daniels, Michael J., additional, Triplett, William T., additional, Rooney, William D., additional, Lott, Donovan J., additional, Forbes, Sean C., additional, Wang, Dah-Jyuu, additional, Senesac, Claudia R., additional, Harrington, Ann T., additional, Finkel, Richard S., additional, Russman, Barry S., additional, Byrne, Barry J., additional, Tennekoon, Gihan I., additional, Walter, Glenn A., additional, Sweeney, H. Lee, additional, and Vandenborne, Krista, additional

Published
2018
Full Text
View/download PDF

43. Chemical shift-based MRI to measure fat fractions in dystrophic skeletal muscle

Author

Triplett, William T., Baligand, Celine, Forbes, Sean C., Willcocks, Rebecca J., Lott, Donovan J., DeVos, Soren, Pollaro, Jim, Rooney, William D., Sweeney, H. Lee, Bönnemann, Carsten, Wang, Dah-Jyuu, Vandenborne, Krista, and Walter, Glenn A.

Subjects
Adult, Collagen Type IV, Male, Magnetic Resonance Spectroscopy, Adolescent, Phantoms, Imaging, Middle Aged, Signal-To-Noise Ratio, Magnetic Resonance Imaging, Article, Muscular Dystrophy, Duchenne, Imaging, Three-Dimensional, Adipose Tissue, Case-Control Studies, Child, Preschool, Image Processing, Computer-Assisted, Humans, Female, Child
Abstract

The relationship between fat fractions (FFs) determined based on multiple TE, unipolar gradient echo images and (1) H magnetic resonance spectroscopy (MRS) was evaluated using different models for fat-water decomposition, signal-to-noise ratios, and excitation flip angles.A combination of single-voxel proton spectroscopy ((1) H-MRS) and gradient echo imaging was used to determine muscle FFs in both normal and dystrophic muscles. In order to cover a large range of FFs, the soleus and vastus lateralis muscles of 22 unaffected control subjects, 16 subjects with collagen VI deficiency (COL6), and 71 subjects with Duchenne muscular dystrophy (DMD) were studied. (1) H-MRS-based FF were corrected for the increased muscle (1) H2 O T1 and T2 values observed in dystrophic muscles.Excellent agreement was found between coregistered FFs derived from gradient echo images fit to a multipeak model with noise bias correction and the relaxation-corrected (1) H-MRS FFs (y = 0.93x + 0.003; R(2) = 0.96) across the full range of FFs. Relaxation-corrected (1) H-MRS FFs and imaging-based FFs were significantly elevated (P0.01) in the muscles of COL6 and DMD subjects.FFs, T2 , and T1 were all sensitive to muscle involvement in dystrophic muscle. MRI offered an additional advantage over single-voxel spectroscopy in that the tissue heterogeneity in FFs could be readily determined.

Published
2013

45. Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys with Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study

Author

Forbes, Sean C., primary, Willcocks, Rebecca J., additional, Triplett, William T., additional, Rooney, William D., additional, Lott, Donovan J., additional, Wang, Dah-Jyuu, additional, Pollaro, Jim, additional, Senesac, Claudia R., additional, Daniels, Michael J., additional, Finkel, Richard S., additional, Russman, Barry S., additional, Byrne, Barry J., additional, Finanger, Erika L., additional, Tennekoon, Gihan I., additional, Walter, Glenn A., additional, Sweeney, H. Lee, additional, and Vandenborne, Krista, additional

Published
2014
Full Text
View/download PDF

47. Skeletal Muscles of Ambulant Children with Duchenne Muscular Dystrophy: Validation of Multicenter Study of Evaluation with MR Imaging and MR Spectroscopy

Author

Forbes, Sean C., primary, Walter, Glenn A., additional, Rooney, William D., additional, Wang, Dah-Jyuu, additional, DeVos, Soren, additional, Pollaro, Jim, additional, Triplett, William, additional, Lott, Donovan J., additional, Willcocks, Rebecca J., additional, Senesac, Claudia, additional, Daniels, Michael J., additional, Byrne, Barry J., additional, Russman, Barry, additional, Finkel, Richard S., additional, Meyer, James S., additional, Sweeney, H. Lee, additional, and Vandenborne, Krista, additional

Published
2013
Full Text
View/download PDF

48. Chemical shift-based MRI to measure fat fractions in dystrophic skeletal muscle

Author

Triplett, William T., primary, Baligand, Celine, additional, Forbes, Sean C., additional, Willcocks, Rebecca J., additional, Lott, Donovan J., additional, DeVos, Soren, additional, Pollaro, Jim, additional, Rooney, William D., additional, Sweeney, H. Lee, additional, Bönnemann, Carsten G., additional, Wang, Dah-Jyuu, additional, Vandenborne, Krista, additional, and Walter, Glenn A., additional

Published
2013
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results