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The consensus was derived using a modified Delphi methodology that involved an exploration phase and then an evaluation phase.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>The exploration phase involved 90-minute virtual 1:1 interviews of 12 paediatric neurologists who cared for 30\u2013120 DMD patients each and had patient contact every one or two weeks. The respondents managed one to ten nmDMD patients taking ataluren. The Discussion Guide for the interviews can be viewed as Appendix A. Following the exploration phase interviews, the interview transcripts were analysed by an independent party to identify common themes, views and opinions and developed 43 draft statements that the Steering Group (authors) reviewed, refined and endorsed a final list of 42 statements. Details of the recruitment of participants for the exploration and evaluation phases can be found under the Methods section.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>A consensus was agreed (&gt;\u200966% of respondents agreeing) for 41 of the 42 statements using results from a consensus survey of healthcare professionals (<jats:italic>n<\/jats:italic>\u2009=\u200920) experienced in the treatment of nmDMD.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>The statements with a high consensus suggest that treatment with ataluren should be initiated as soon as possible to delay disease progression and allow patients to remain ambulatory for as long as possible. Ataluren is indicated for the treatment of Duchenne muscular dystrophy that results from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older (see Summary of Product Characteristics for each country)<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s12883-024-03570-x","type":"journal-article","created":{"date-parts":[[2024,2,21]],"date-time":"2024-02-21T07:02:21Z","timestamp":1708498941000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["The role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutation duchenne muscular dystrophy - a consensus derived using a modified Delphi methodology in Eastern Europe, Greece, Israel and Sweden"],"prefix":"10.1186","volume":"24","author":[{"given":"Tanja","family":"Golli","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lenka","family":"Ju\u0159\u00edkov\u00e1","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Sejersen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig","family":"Dixon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,2,21]]},"reference":[{"key":"3570_CR1","doi-asserted-by":"publisher","first-page":"291","DOI":"10.1152\/physrev.00028.2001","volume":"82","author":"DJ Blake","year":"2002","unstructured":"Blake DJ, Weir A, Newey SE, Davies. 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Interviews with healthcare professionals do not require ethics committee approval and no identifiable patients were discussed during the interviews. The 12 clinician interviews were conducted in line with all relevant codes of conduct including ABPI, Data Protection Act, Market Research Society, British Healthcare Business Intelligence Association (BHBIA). The 12 clinicians that were interviewed were provided with a contract outlining all the relevant permissions and code of conducts, a signed contract was in place with all 12 clinicians prior to the interview being conducted. There was no direct contact with the clinicians (<i>n<\/i>\u2009+\u200920) who completed the survey of the evaluation phase.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"The 12 clinicians that were interviewed were provided with a contract outlining all the relevant permissions and code of conducts, a signed contract was in place with all 12 clinicians prior to the interview being conducted. The data collected and to be published is anonymous.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The Steering Group and experts interviewed as part of the exploration phase were paid an honorarium at fair market value for their time. The clinician respondents to the evaluation phase were also paid a modest honorarium for the time taken to complete the questionnaire. Tanja Golli: Recipient of honoraria for lectures from Novartis and PTC Therapeutics. Lenka Ju\u0159\u00edkov\u00e1: Recipient of honoraria received for lectures from PTC Therapeutics, Biogen and Sanofi Genzyme. Thomas Sejersen: Recipient of honoraria received for lectures or consultancy from Biogen, Novartis, PTC Therapeutics, Sarepta Therapeutics, Roche, Hansa Biopharma and Sanofi Genzyme. Craig Dixon: Funding from PTC Therapeutics to conduct the consensus project.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"73"}}