A survey of the feasibility of developing osteoporosis clinical trials in Duchenne muscular dystrophy: Survey of the opinion of young people with Duchenne muscular dystrophy, families and clinicians.
作者： Sze Choong Wong , Shuko Joseph , Nadia Capaldi , Marina Di Marco , Jennifer Dunne
关键词: Limited evidence 、 Osteoporosis 、 Muscular dystrophy 、 Clinical trial 、 Pediatrics 、 Duchenne muscular dystrophy 、 Bone density 、 Medicine
摘要: Background/aimsGiven the extent of osteoporosis in people with Duchenne muscular dystrophy treated glucocorticoids and limited evidence bone-protective therapies, clinical trials are ne...
sagepub.com
sci-hub.st 参考文章(23)
Ilene L. Hollin, Holly L. Peay, John F. P. Bridges, Caregiver Preferences for Emerging Duchenne Muscular Dystrophy Treatments: A Comparison of Best-Worst Scaling and Conjoint Analysis The Patient: Patient-Centered Outcomes Research. ,vol. 8, pp. 19- 27 ,(2015) , 10.1007/S40271-014-0104-X
Deborah J. Bowen, Matthew Kreuter, Bonnie Spring, Ludmila Cofta-Woerpel, Laura Linnan, Diane Weiner, Suzanne Bakken, Cecilia Patrick Kaplan, Linda Squiers, Cecilia Fabrizio, Maria Fernandez, How We Design Feasibility Studies American Journal of Preventive Medicine. ,vol. 36, pp. 452- 457 ,(2009) , 10.1016/J.AMEPRE.2009.02.002
Holly L. Peay, Ilene Hollin, Ryan Fischer, John F.P. Bridges, A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy Clinical Therapeutics. ,vol. 36, pp. 624- 637 ,(2014) , 10.1016/J.CLINTHERA.2014.04.011
Catrin Tudur Smith, Paula R. Williamson, Michael W. Beresford, Methodology of clinical trials for rare diseases. Best Practice & Research: Clinical Rheumatology. ,vol. 28, pp. 247- 262 ,(2014) , 10.1016/J.BERH.2014.03.004
Roxanna M. Bendixen, Lauren P. Morgenroth, Kristin L. Clinard, Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy. Clinical Therapeutics. ,vol. 38, pp. 1474- 1484 ,(2016) , 10.1016/J.CLINTHERA.2016.04.001
S. Joseph, S. McCarrison, S.C. Wong, Skeletal Fragility in Children with Chronic Disease. Hormone Research in Paediatrics. ,vol. 86, pp. 71- 82 ,(2016) , 10.1159/000447583
L. M. Ward, J. Ma, H. J. McMillan, G. Karagüzel, C. Goodin, J. Wasson, M. A. Matzinger, P. DesClouds, D. Cram, M. Page, V. N. Konji, B. Lentle, The time to and determinants of first fractures in boys with Duchenne muscular dystrophy Osteoporosis International. ,vol. 28, pp. 597- 608 ,(2017) , 10.1007/S00198-016-3774-5
Mabrouka A Altowati, Ashley P Jones, Helen Hickey, Paula R Williamson, Farah M Barakat, Nicolene C Plaatjies, Ben Hardwick, Richard K Russell, Thomas Jaki, S Faisal Ahmed, Ian R Sanderson, None, Assessing the feasibility of injectable growth-promoting therapy in Crohn's disease. Pilot and Feasibility Studies. ,vol. 2, pp. 71- 71 ,(2016) , 10.1186/S40814-016-0112-9
Brenda L. Wong, Irina Rybalsky, Karen C. Shellenbarger, Cuixia Tian, Mary A. McMahon, Meilan M. Rutter, Hemant Sawnani, John L. Jefferies, Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment The Journal of Pediatrics. ,vol. 182, pp. 296- 303 ,(2017) , 10.1016/J.JPEDS.2016.11.078
Janet Woodcock, Lisa M. LaVange, Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both The New England Journal of Medicine. ,vol. 377, pp. 62- 70 ,(2017) , 10.1056/NEJMRA1510062