{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T18:09:47Z","timestamp":1791828587125,"version":"4.3.4"},"reference-count":14,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2019,11,13]],"date-time":"2019-11-13T00:00:00Z","timestamp":1573603200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013699","name":"Ohio State University Comprehensive Cancer Center \u2013 Arthur G. James Cancer Hospital and Richard J. 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A final shortlist of 22 questions was discussed during an international multi\u2010stakeholder workshop, and a consensus was reached on the top 11 priorities using an adapted nominal group technique.In the first survey, a total of 1388 questions were identified from 763 responders from 58 countries. These original uncertainties were refined into 72 summary questions for a second prioritization survey. In total 562 responders from 58 countries answered the second survey. From the second survey, the top 10 for patients, carers and healthcare professionals was identified and this shortlist of 22 questions was taken to the final workshop. During the final workshop, participants identified the worldwide top 11 research priorities for liver GSD. In addition, a top three research priorities per liver GSD subtype was identified.This unique priority setting partnership is the first international, multilingual priority setting partnership focusing on ultra\u2010rare diseases. This process provides a valuable resource for researchers and funding agencies to foster interdisciplinary and transnational research projects with a clear benefit for patients.<\/jats:p>","DOI":"10.1002\/jimd.12178","type":"journal-article","created":{"date-parts":[[2019,10,6]],"date-time":"2019-10-06T08:01:06Z","timestamp":1570348866000},"page":"279-289","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":45,"title":["Research priorities for liver glycogen storage disease: An international priority setting partnership with the James Lind Alliance"],"prefix":"10.1002","volume":"43","author":[{"given":"Fabian","family":"Peeks","sequence":"first","affiliation":[{"name":"Section of Metabolic Diseases, Beatrix Children's Hospital University Medical Center Groningen, University of Groningen  Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Willemijn F.","family":"Boonstra","sequence":"additional","affiliation":[{"name":"Section of Metabolic Diseases, Beatrix Children's Hospital University Medical Center Groningen, University of Groningen  Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lut","family":"de Baere","sequence":"additional","affiliation":[{"name":"Belgische Organisatie voor Kinderen en volwassenen met een Stofwisselingsziekte VZW  Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camilla","family":"Car\u00f8e","sequence":"additional","affiliation":[{"name":"Paediatric Nutrition, University Hospital of Copenhagen Rigshospitalet  Copenhagen Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Casswall","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology Hepatology and Nutrition, Karolinska University Hospital"},{"name":"CLINTEC, Karolinska Institutet  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dami\u00e1n","family":"Cohen","sequence":"additional","affiliation":[{"name":"Glucolatino, Rosario  Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine","family":"Cowan","sequence":"additional","affiliation":[{"name":"James Lind Alliance University of Southampton  Southampton UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Iris","family":"Ferrecchia","sequence":"additional","affiliation":[{"name":"Glycogen Storage Disease Program University of Connecticut  Farmington Connecticut"},{"name":"Connecticut Children's Medical Center  Hartford Connecticut"},{"name":"Association for Glycogen Storage Disease"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Ferriani","sequence":"additional","affiliation":[{"name":"Associa\u00e7\u00e3o Brasileira de Glicogenose (ABGLICO)  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Gimbert","sequence":"additional","affiliation":[{"name":"L'Association Francophone des Glycog\u00e9noses  France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcus","family":"Landgren","sequence":"additional","affiliation":[{"name":"Scandinavian Association for Glycogen Storage Disease (SAGSD)  Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nerea L.","family":"Maldonado","sequence":"additional","affiliation":[{"name":"Asociaci\u00f3n Espa\u00f1ola de Enfermos de Glucogenosis, General Practitioner Institut Catal\u00e0 de la Salut (ICS)  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"McMillan","sequence":"additional","affiliation":[{"name":"Association for Glycogen Storage Disease  UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antal","family":"Nemeth","sequence":"additional","affiliation":[{"name":"Department of Pediatric Gastroenterology Hepatology and Nutrition, Karolinska University Hospital"},{"name":"CLINTEC, Karolinska Institutet  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niccol\u00f2","family":"Seidita","sequence":"additional","affiliation":[{"name":"Associazione Italiana Glicogenosi  Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ute","family":"Stachelhaus\u2010Theimer","sequence":"additional","affiliation":[{"name":"Selbsthilfegruppe Glykogenose Deutschland e.V.  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