Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel

https://m2.mtmt.hu/ eng 2026-10-24 21:05 JournalArticle 35092369 VALIDATED true Funding Agency and Grant Number: PTC Therapeutics Funding text: The authors would like to thank Jenny Hepburn from MASS Team for assistance with the exploration phase and data analysis, and A-Z Medical Writing Ltd for their assistance with the preparation of this manuscript.DAS:All data supporting the findings of this study are available within the paper and its Supplementary Information, including the raw and analysed data from the evaluation phase. 0 false 2026-10-24T17:59:03.453+0000 2024-07-23T06:55:29.088+0000 2024-07-10T05:30:08.458+0000 true 10015544 Csajbók Edit /api/admin/10015544 Admin Edit Csajbók (SE_KK_Admin5_CSE, admin) true 10015544 2024-07-29T16:46:57.656+0000 2024-07-29T16:46:57.656+0000 2024-07-10T05:37:45.872+0000 true 10015544 Csajbók Edit /api/admin/10015544 Admin Edit Csajbók (SE_KK_Admin5_CSE, admin) true 10015544 true false true 24 24 /api/publicationtype/24 PublicationType Journal Article 1 true 24 JournalArticle true 10000059 Article true 24 24 /api/publicationtype/24 PublicationType Journal Article 1 true 24 JournalArticle /api/subtype/10000059 Szakcikk SubType Article (Journal Article) 101 true 10000059 true 1 /api/category/1 Category Scientific true 1 Molnar, Maria Judit Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel true true /api/journal/10007208 REVIEWED ORPHANET JOURNAL OF RARE DISEASES 1750-1172 1750-1172 true false 10007208 false 10007208 true 1750-1172 1750-1172 Journal FOREIGN 19 1 260 260 13 2024 An increasing number of patients with Duchenne muscular dystrophy (DMD) now have access to improved standard of care and disease modifying treatments, which improve the clinical course of DMD and extend life expectancy beyond 30 years of age. A key issue for adolescent DMD patients is the transition from paediatric- to adult-oriented healthcare. Adolescents and adults with DMD have unique but highly complex healthcare needs associated with long-term steroid use, orthopaedic, respiratory, cardiac, psychological, and gastrointestinal problems meaning that a comprehensive transition process is required. A sub-optimal transition into adult care can have disruptive and deleterious consequences for a patient’s long-term care. This paper details the results of a consensus amongst clinicians on transitioning adolescent DMD patients from paediatric to adult neurologists that can act as a guide to best practice to ensure patients have continuous comprehensive care at every stage of their journey. 2024 true true true false false GOLD 2026-10-24 false http://www.ojrd.com/ 5 0 5 5 0 5 5 5 5 5 5 5 5 5 0 0 5 5 5 5 0 1 1 Folyóiratcikk Journal Article 24 5 Könyvrészlet Chapter in Book 25 0 Könyv Book 23 0 Egyéb konferenciaközlemény Conference paper 31 0 Egyéb konferenciakötet Conference proceedings 32 0 Oltalmi formák Protection forms 26 0 Disszertáció Thesis 28 0 Egyéb Miscellaneous 29 0 Alkotás Achievement 22 0 Kutatási adat Research data 33 0 Folyóiratcikk Journal Article 24 0 0 0 Könyvrészlet Chapter in Book 25 0 0 0 Könyv Book 23 0 0 0 Egyéb konferenciaközlemény Conference paper 31 0 0 0 Egyéb konferenciakötet Conference proceedings 32 0 0 0 Oltalmi formák Protection forms 26 0 0 0 Disszertáció Thesis 28 0 0 0 Egyéb Miscellaneous 29 0 0 0 Alkotás Achievement 22 0 0 0 Kutatási adat Research data 33 0 0 0 2025 0 2 2 2 2 2026 0 3 3 3 3 Q1 false 10023270 false false false 2024-07-09T05:37:45.765+0000 Genomikai Medicina és Ritka Betegségek Intézete (SE / AOK / I); Gyermekgyógyászati Klinika (SE / AOK / K) 2 17 2 11 0 true Language 10002 /api/language/10002 English Angol English true 2 true PersonAuthorship 118192657 /api/authorship/118192657 Molnar, Maria Judit [Molnár, Mária Judit (Neurológia), author] Genomic Medicine and the Institute of Rare Dise... (SU / FM / I) 1 0.091 true false false Author 10002781 /api/author/10002781 Mária Judit Molnár (Neurológia) Molnár Mária Judit true 10002781 true Molnar Maria Judit true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192658 /api/authorship/118192658 Szabó, Léna [Szabó, Léna (Gyermekgyógyászat), author] Department of Pediatrics (SU / FM / C) 2 0.091 false false false Author 10023270 /api/author/10023270 Léna Szabó (Gyermekgyógyászat) Szabó Léna true 10023270 true Szabó Léna true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192659 /api/authorship/118192659 Vladacenco, Oana Aurelia 3 0.09090909 false false false Vladacenco Oana Aurelia true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192660 /api/authorship/118192660 Cobzaru, Ana Maria 4 0.09090909 false false false Cobzaru Ana Maria true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192661 /api/authorship/118192661 Dor, Talya 5 0.09090909 false false false Dor Talya true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192662 /api/authorship/118192662 Dori, Amir 6 0.09090909 false false false Dori Amir true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192663 /api/authorship/118192663 Papadimas, Georgios 7 0.09090909 false false false Papadimas Georgios true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192664 /api/authorship/118192664 Juříková, Lenka 8 0.09090909 false false false Juříková Lenka true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192665 /api/authorship/118192665 Litvinenko, Ivan 9 0.09090909 false false false Litvinenko Ivan true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192666 /api/authorship/118192666 Tournev, Ivailo 10 0.09090909 false false false Tournev Ivailo true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PersonAuthorship 118192667 /api/authorship/118192667 Dixon, Craig ✉ 11 0.091 false true true Dixon Craig true false false AuthorshipType 1 /api/authorshiptype/1 Author 0 true 0 true false true PublicationIdentifier 26779513 /api/publicationidentifier/26779513 DOI: 10.1186/s13023-024-03270-2 PlainSource 6 /api/publicationsource/6 DOI PublicationSourceType 10001 /api/publicationsourcetype/10001 DOI true true true DOI DOI https://doi.org/@@@ true true 6 true NONE IDENTICAL 10.1186/s13023-024-03270-2 https://doi.org/10.1186/s13023-024-03270-2 false true PublicationIdentifier 26856539 /api/publicationidentifier/26856539 WoS: 001265783100001 PlainSource 1 /api/publicationsource/1 WoS PublicationSourceType 10003 /api/publicationsourcetype/10003 Indexelő adatbázis false true true WoS WoS https://www.webofscience.com/wos/woscc/full-record/@@@ true true 1 true IDENTICAL 001265783100001 https://www.webofscience.com/wos/woscc/full-record/001265783100001 true true PublicationIdentifier 26856540 /api/publicationidentifier/26856540 Scopus: 85198124341 PlainSource 3 /api/publicationsource/3 Scopus PublicationSourceType 10003 /api/publicationsourcetype/10003 Indexelő adatbázis false true true Scopus Scopus http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-@@@ true true 3 true IDENTICAL 85198124341 http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-85198124341 true true PublicationIdentifier 26856541 /api/publicationidentifier/26856541 PubMed: 38982500 PlainSource 17 /api/publicationsource/17 PubMed PublicationSourceType 10003 /api/publicationsourcetype/10003 Indexelő adatbázis false true true PubMed PubMed http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=@@@&dopt=Abstract true true 17 true IDENTICAL 38982500 http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=38982500&dopt=Abstract true true SjrRating 11466290 /api/sjrrating/11466290 sjr:Q1 (2024) Scopus - Genetics (clinical) ORPHANET JOURNAL OF RARE DISEASES 1750-1172 1750-1172 23 0.24 journal RatingType 10002 /api/ratingtype/10002 sjr sjr true true ClassificationExternal 2716 /api/classificationexternal/2716 Scopus - Genetics (clinical) true 2716 true Q1 DIRECT true true /api/publication/35092369 Molnar Maria Judit et al. Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel. (2024) ORPHANET JOURNAL OF RARE DISEASES 1750-1172 1750-1172 19 1<div class="JournalArticle Publication long-list"> <div class="authors"> <img title="Forrásközlemény" style="float: left" src="/frontend/resources/grid/publication-core-icon.png"> <img title="Idézőközlemény" style="float: left" src="/frontend/resources/grid/publication-citation-icon.png"> <div class="autype autype0"> <span class="author-name" mtid="10002781"><a href="/gui2/?type=authors&mode=browse&sel=10002781" target="_blank">Molnar Maria Judit (<span class="authorship-author-name">Molnár Mária Judit</span> <span class="authorAux-mtmt"> Neurológia</span>) </a> </span> <span class="author-affil"><span title="Semmelweis University">SU</span>/<span title="Faculty of Medicine">FM</span>/<span title="Instituties">I</span>/Genomic Medicine and the Institute of Rare Diseases</span> ;    <span class="author-name" mtid="10023270"><a href="/gui2/?type=authors&mode=browse&sel=10023270" target="_blank">Szabó Léna (<span class="authorship-author-name">Szabó Léna</span> <span class="authorAux-mtmt"> Gyermekgyógyászat</span>) </a> </span> <span class="author-affil"><span title="Semmelweis University">SU</span>/<span title="Faculty of Medicine">FM</span>/<span title="Clinical">C</span>/Department of Pediatrics</span> ;    <span class="author-name" >Vladacenco Oana Aurelia </span> ;    <span class="author-name" >Cobzaru Ana Maria </span> ;    <span class="author-name" >Dor Talya </span> ;    <span class="author-name" >Dori Amir </span> ;    <span class="author-name" >Papadimas Georgios </span> ;    <span class="author-name" >Juříková Lenka </span> ;    <span class="author-name" >Litvinenko Ivan </span> ;    <span class="author-name" >Tournev Ivailo </span> ;    <span class="author-name" >Dixon Craig ✉ </span> </div> </div> <div class="title"><a href="/gui2/?mode=browse&params=publication;35092369" target="_blank">Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel</a></div> <div> <span class="journal-title">ORPHANET JOURNAL OF RARE DISEASES</span> <span class="journal-issn">(<a target="_blank" href="https://portal.issn.org/resource/ISSN/1750-1172">1750-1172</a> <a target="_blank" href="https://portal.issn.org/resource/ISSN/1750-1172">1750-1172</a>)</span>: <span class="journal-volume">19</span> <span class="journal-issue">1</span> <span class="page"> Paper 260. 13 p. </span> <span class="year">(2024)</span> </div> <div class="pub-footer"> <span class="language" xmlns="http://www.w3.org/1999/html">Language: English | </span> <span class="identifiers"> <span class="id identifier oa_NONE" title=" None "> <a style="color:blue" title="10.1186/s13023-024-03270-2" target="_blank" href="https://doi.org/10.1186/s13023-024-03270-2"> DOI </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="001265783100001" target="_blank" href="https://www.webofscience.com/wos/woscc/full-record/001265783100001"> WoS </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="85198124341" target="_blank" href="http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-85198124341"> Scopus </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="38982500" target="_blank" href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=38982500&dopt=Abstract"> PubMed </a> </span> </span> <OnlyViewableByAuthor><div class="ratings"> <div class="journal-subject">Journal subject: Scopus - Genetics (clinical)   Rank: Q1</div> <div class="journal-subject">Journal subject: Scopus - Medicine (miscellaneous)   Rank: Q1</div> <div class="journal-subject">Journal subject: Scopus - Pharmacology (medical)   Rank: Q1</div> </div></OnlyViewableByAuthor> <div class="publication-citation" style="margin-left: 0.5cm;"> <span title="" class="citingPub-count">Citing papers: 5</span> | Independent citation: 5 | Self citation: 0 | Unknown citation: 0 | Number of citations in WoS: 5 | Number of citations in Scopus: 5 | WoS/Scopus assigned: 5 | Number of citations with DOI: 5 </div> <div class="publication-citation"> <a target="_blank" href="/api/publication?cond=citations.related;eq;35092369&sort=publishedYear,desc&sort=title"> Number of cited publications: 1 </a> </div> <div class="mtid"><span class="long-pub-mtid">Publication: 35092369</span> | <span class="status-data status-VALIDATED"> Validated </span> Core Citing | <span class="type-subtype">Journal Article ( Article ) </span> | <span class="pub-category">Scientific</span> | <span class="publication-sourceOfData">RIS-Egyéb</span> </div> <div class="notDuplums"><a target="_blank" href="/api/publication/35092369/notduplums">1 marked as not duplum</a> </div> <div class="lastModified">Last Modified: 2024.07.23. 08:55 Márta Molnár-Taga (SE 4-es admin) </div> <pre class="comment" style="margin-top: 0; margin-bottom: 0;"><u>Comments</u>: Funding Agency and Grant Number: PTC Therapeutics Funding text: The authors would like to thank Jenny Hepburn from MASS Team for assistance with the exploration phase and data analysis, and A-Z Medical Writing Ltd for their assistance with the preparation of this manuscript.DAS:All data supporting the findings of this study are available within the paper and its Supplementary Inform...</pre> </div></div>