{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T15:07:09Z","timestamp":1791558429703,"version":"4.3.4"},"reference-count":42,"publisher":"Public Library of Science (PLoS)","issue":"5","license":[{"start":{"date-parts":[[2018,5,17]],"date-time":"2018-05-17T00:00:00Z","timestamp":1526515200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health (US)","doi-asserted-by":"crossref","award":["T32-GM008638"],"award-info":[{"award-number":["T32-GM008638"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0197513","type":"journal-article","created":{"date-parts":[[2018,5,17]],"date-time":"2018-05-17T13:34:05Z","timestamp":1526564045000},"page":"e0197513","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":68,"title":["Mitochondrial disease patient motivations and barriers to participate in clinical trials"],"prefix":"10.1371","volume":"13","author":[{"given":"Zarazuela","family":"Zolkipli-Cunningham","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rui","family":"Xiao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Stoddart","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth M.","family":"McCormick","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Holberts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie","family":"Burrill","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shana","family":"McCormack","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren","family":"Williams","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John L. P.","family":"Thompson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1723-6728","authenticated-orcid":true,"given":"Marni J.","family":"Falk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2018,5,17]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"16080","DOI":"10.1038\/nrdp.2016.80","article-title":"Mitochondrial diseases","volume":"2","author":"GS Gorman","year":"2016","journal-title":"Nat Rev Dis Primers"},{"issue":"5","key":"ref2","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1002\/ana.24362","article-title":"Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease","volume":"77","author":"GS Gorman","year":"2015","journal-title":"Ann Neurol"},{"issue":"1\u20132","key":"ref3","doi-asserted-by":"crossref","first-page":"70","DOI":"10.1016\/0022-510X(90)90015-F","article-title":"Ubidecarenone in the treatment of mitochondrial myopathies: a multi-center double-blind trial","volume":"100","author":"N Bresolin","year":"1990","journal-title":"J Neurol Sci"},{"key":"ref4","first-page":"CD004426","article-title":"Treatment for mitochondrial disorders","volume":"4","author":"G Pfeffer","year":"2012","journal-title":"Cochrane Database Syst Rev"},{"issue":"8","key":"ref5","doi-asserted-by":"crossref","first-page":"474","DOI":"10.1038\/nrneurol.2013.129","article-title":"New treatments for mitochondrial disease-no time to drop our standards","volume":"9","author":"G Pfeffer","year":"2013","journal-title":"Nat Rev Neurol"},{"issue":"5","key":"ref6","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1016\/j.mito.2011.05.002","article-title":"Why are there no proven therapies for genetic mitochondrial diseases?","volume":"11","author":"PW Stacpoole","year":"2011","journal-title":"Mitochondrion"},{"issue":"Suppl","key":"ref7","doi-asserted-by":"crossref","first-page":"S136","DOI":"10.1016\/j.mito.2007.03.008","article-title":"The evidence basis for coenzyme Q therapy in oxidative phosphorylation disease","volume":"7","author":"RH Haas","year":"2007","journal-title":"Mitochondrion"},{"issue":"13\u201314","key":"ref8","doi-asserted-by":"crossref","first-page":"1561","DOI":"10.1016\/j.addr.2008.05.001","article-title":"The mitochondrial cocktail: rationale for combined nutraceutical therapy in mitochondrial cytopathies","volume":"60","author":"MA Tarnopolsky","year":"2008","journal-title":"Adv Drug Deliv Rev"},{"issue":"3","key":"ref9","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1016\/j.ymgme.2009.11.005","article-title":"Treatment of mitochondrial electron transport chain disorders: a review of clinical trials over the past decade","volume":"99","author":"DS Kerr","year":"2010","journal-title":"Mol Genet Metab"},{"issue":"6","key":"ref10","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1007\/s11940-009-0046-0","article-title":"A modern approach to the treatment of mitochondrial disease","volume":"11","author":"S Parikh","year":"2009","journal-title":"Curr Treat Options Neurol"},{"issue":"9","key":"ref11","doi-asserted-by":"crossref","first-page":"689","DOI":"10.1038\/gim.2014.177","article-title":"Diagnosis and management of mitochondrial disease: a consensus statement from the Mitochondrial Medicine Society","volume":"17","author":"S Parikh","year":"2015","journal-title":"Genet Med"},{"issue":"5","key":"ref12","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1097\/WCO.0000000000000243","article-title":"Duchenne muscular dystrophy: clinical trials and emerging tribulations","volume":"28","author":"PB Shieh","year":"2015","journal-title":"Curr Opin Neurol"},{"key":"ref13","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1186\/s12883-015-0408-z","article-title":"Improving clinical trial design for Duchenne muscular dystrophy","volume":"15","author":"L Merlini","year":"2015","journal-title":"BMC Neurol"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1186\/1750-1172-8-44","article-title":"SMA- EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe","volume":"8","author":"N Kayadjanian","year":"2013","journal-title":"Orphanet J Rare Dis"},{"issue":"3","key":"ref15","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1517\/14728214.2015.1041375","article-title":"Moving towards treatments for spinal muscular atrophy: hopes and limits","volume":"20","author":"B Wirth","year":"2015","journal-title":"Expert Opin Emerg Drugs"},{"issue":"2","key":"ref16","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1016\/j.cct.2013.04.004","article-title":"Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates","volume":"35","author":"EA de Blieck","year":"2013","journal-title":"Contemp Clin Trials"},{"key":"ref17","unstructured":"FDA. Rare Diseases: Common Issues in Drug Development Guidance for Industry. 2015."},{"issue":"2","key":"ref18","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1007\/s13311-012-0174-1","article-title":"Molecular genetic testing for mitochondrial disease: from one generation to the next","volume":"10","author":"E McCormick","year":"2013","journal-title":"Neurotherapeutics"},{"issue":"1","key":"ref19","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.ymgme.2007.11.018","article-title":"The in-depth evaluation of suspected mitochondrial disease","volume":"94","author":"RH Haas","year":"2008","journal-title":"Mol Genet Metab"},{"issue":"Pt 2","key":"ref20","doi-asserted-by":"crossref","first-page":"e11","DOI":"10.1093\/brain\/aww303","article-title":"Treatable mitochondrial diseases: cofactor metabolism and beyond","volume":"140","author":"F Distelmaier","year":"2017","journal-title":"Brain"},{"issue":"4","key":"ref21","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1016\/j.cct.2012.02.012","article-title":"The Rare Diseases Clinical Research Network Contact Registry update: features and functionality","volume":"33","author":"RL Richesson","year":"2012","journal-title":"Contemp Clin Trials"},{"issue":"1","key":"ref22","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1186\/s13023-016-0445-8","article-title":"The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network","volume":"11","author":"PA Merkel","year":"2016","journal-title":"Orphanet J Rare Dis"},{"key":"ref23","unstructured":"FDA. Critical Path Innovation Meeting Regarding Drug Development for Mitochondrial Diseases. 2015."},{"issue":"4","key":"ref24","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1016\/j.jval.2013.04.004","article-title":"Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force","volume":"16","author":"LS Matza","year":"2013","journal-title":"Value Health"},{"issue":"9","key":"ref25","doi-asserted-by":"crossref","first-page":"1130","DOI":"10.1177\/0883073810371509","article-title":"Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy","volume":"25","author":"CM McDonald","year":"2010","journal-title":"J Child Neurol"},{"issue":"11","key":"ref26","doi-asserted-by":"crossref","first-page":"1711","DOI":"10.1212\/01.wnl.0000218155.46739.90","article-title":"Measuring Friedreich ataxia: complementary features of examination and performance measures","volume":"66","author":"DR Lynch","year":"2006","journal-title":"Neurology"},{"issue":"8","key":"ref27","doi-asserted-by":"crossref","first-page":"698","DOI":"10.1111\/dmcn.12119","article-title":"Towards the harmonization of outcome measures in children with mitochondrial disorders","volume":"55","author":"S Koene","year":"2013","journal-title":"Dev Med Child Neurol"},{"issue":"3","key":"ref28","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/j.ymgme.2016.09.002","article-title":"Nutritional interventions in primary mitochondrial disorders: Developing an evidence base","volume":"119","author":"KM Camp","year":"2016","journal-title":"Mol Genet Metab"},{"issue":"2","key":"ref29","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1007\/s13311-013-0176-7","article-title":"Review of clinical trials for mitochondrial disorders: 1997\u20132012","volume":"10","author":"DS Kerr","year":"2013","journal-title":"Neurotherapeutics"},{"issue":"2","key":"ref30","doi-asserted-by":"crossref","first-page":"161","DOI":"10.2217\/pme.11.7","article-title":"The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?","volume":"8","author":"EO Lillie","year":"2011","journal-title":"Per Med"},{"issue":"5","key":"ref31","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1177\/0883073813481622","article-title":"Mitochondrial disease: a challenge for the caregiver, the family, and society","volume":"28","author":"K Sofou","year":"2013","journal-title":"J Child Neurol"},{"issue":"5","key":"ref32","doi-asserted-by":"crossref","first-page":"531","DOI":"10.1093\/ckj\/sfv060","article-title":"Understanding the physical and emotional impact of early-stage ADPKD: experiences and perspectives of patients and physicians","volume":"8","author":"A Baker","year":"2015","journal-title":"Clin Kidney J"},{"key":"ref33","article-title":"\u2019A short time but a lovely little short time\u2019: Bereaved parents\u2019 experiences of having a child with spinal muscular atrophy type 1","author":"EJ Higgs","year":"2015","journal-title":"J Paediatr Child Health"},{"issue":"1","key":"ref34","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1016\/j.mito.2012.11.002","article-title":"Developing outcome measures for pediatric mitochondrial disorders: which complaints and limitations are most burdensome to patients and their parents?","volume":"13","author":"S Koene","year":"2013","journal-title":"Mitochondrion"},{"issue":"3","key":"ref35","doi-asserted-by":"crossref","first-page":"134","DOI":"10.4103\/2229-3485.159936","article-title":"Patient centric approach for clinical trials: Current trend and new opportunities","volume":"6","author":"NS Sharma","year":"2015","journal-title":"Perspect Clin Res"},{"key":"ref36","article-title":"Reconciling evidence-based medicine and patient-centred care: defining evidence-based inputs to patient-centred decisions","author":"RR Weaver","year":"2015","journal-title":"J Eval Clin Pract"},{"issue":"5","key":"ref37","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1097\/WCO.0000000000000245","article-title":"What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?","volume":"28","author":"RT Wang","year":"2015","journal-title":"Curr Opin Neurol"},{"issue":"1","key":"ref38","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1378\/chest.13-0606","article-title":"Rare diseases research: expanding collaborative translational research opportunities","volume":"144","author":"SC Groft","year":"2013","journal-title":"Chest"},{"key":"ref39","doi-asserted-by":"crossref","first-page":"579","DOI":"10.1146\/annurev-genom-091212-153525","article-title":"The role of patient advocacy organizations in shaping genomic science","volume":"14","author":"PP Koay","year":"2013","journal-title":"Annu Rev Genomics Hum Genet"},{"issue":"2","key":"ref40","first-page":"302","article-title":"Participation of people with haemophilia in clinical trials of new treatments: an investigation of patients\u2019 motivations and existing barriers","volume":"13","author":"S Henrard","year":"2015","journal-title":"Blood Transfus"},{"issue":"9","key":"ref41","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1016\/j.parkreldis.2015.07.018","article-title":"Recruitment strategies and patient selection in clinical trials for Parkinson\u2019s disease: Going viral and keeping science and ethics at the highest standards","volume":"21","author":"M Picillo","year":"2015","journal-title":"Parkinsonism Relat Disord"},{"key":"ref42","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.cct.2015.11.006","article-title":"&quot;Watching time tick by \u2026&quot;: Decision making for Duchenne muscular dystrophy trials","volume":"46","author":"HL Peay","year":"2016","journal-title":"Contemp Clin Trials"}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0197513","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,6]],"date-time":"2024-07-06T17:10:15Z","timestamp":1720285815000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0197513"}},"subtitle":[],"editor":[{"given":"Hemachandra","family":"Reddy","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2018,5,17]]},"references-count":42,"journal-issue":{"issue":"5","published-online":{"date-parts":[[2018,5,17]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0197513","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,5,17]]}}}