{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,25]],"date-time":"2026-07-25T14:37:24Z","timestamp":1784990244610,"version":"3.55.0"},"reference-count":30,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,5,25]],"date-time":"2023-05-25T00:00:00Z","timestamp":1684972800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["HL135853"],"award-info":[{"award-number":["HL135853"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","award":["HL148681"],"award-info":[{"award-number":["HL148681"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["pericles.pericles-gcp.literatumonline.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The overall burden of disease in persons with haemophilia continues to be high despite the latest advancements in therapeutics. Clinical trials testing prenatal treatments for several genetic disorders\u00a0are underway or are recruiting subjects, attesting to the much\u2010needed change in paradigm of how patients with monogenic disorders can be treated. Here we investigate the overall attitude towards prenatal diagnosis, preferences on types of prenatal therapies for haemophilia, the level of 'acceptable' risk tolerated, and which social and moral pressures or disease personal experiences may predict willingness of individuals to consider foetal therapy in a future pregnancy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A multidisciplinary team designed the survey, and the study was carried out using REDCap, and publicized through the National Haemophilia Foundation. Subjects \u226518 years of age were eligible to participate in the study. We assessed participants\u2019 attitudes towards prenatal therapy and their level of 'acceptable' risk towards the procedure and therapy. The survey was completed by 67 adults, the majority females. Respondents were willing to undergo prenatal diagnosis, and their main concerns related to the well\u2010being of the pregnant woman and the foetus regarding lasting therapeutic efficacy, side effects of the therapy, and procedural risks, but they were likely to accept a wide range of prenatal therapeutic options, particularly if the foetal therapy proved to be long\u2010lasting and safe.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>These data demonstrate the willingness of persons with haemophilia, and the haemophilia community, to explore new treatment options beyond the currently offered approaches.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14805","type":"journal-article","created":{"date-parts":[[2023,5,25]],"date-time":"2023-05-25T17:49:37Z","timestamp":1685036977000},"page":"1024-1031","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Acceptability of prenatal diagnosis and prenatal treatment of haemophilia using cell and gene therapies within US haemophilia community"],"prefix":"10.1111","volume":"29","author":[{"given":"Quan D. M.","family":"Pham","sequence":"first","affiliation":[{"name":"Wake Forest Institute for Regenerative Medicine Fetal Research and Therapy Program Wake Forest School of Medicine (WFSOM)  Winston Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon M.","family":"Thomson","sequence":"additional","affiliation":[{"name":"Wake Forest Institute for Regenerative Medicine Fetal Research and Therapy Program Wake Forest School of Medicine (WFSOM)  Winston Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Burk N.","family":"Schaible","sequence":"additional","affiliation":[{"name":"Center for Research in Obstetrics and Gynecology WFSOM  Winston Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin D.","family":"Mills","sequence":"additional","affiliation":[{"name":"National Hemophilia Foundation  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony","family":"Atala","sequence":"additional","affiliation":[{"name":"Wake Forest Institute for Regenerative Medicine Fetal Research and Therapy Program Wake Forest School of Medicine (WFSOM)  Winston Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher D.","family":"Porada","sequence":"additional","affiliation":[{"name":"Wake Forest Institute for Regenerative Medicine Fetal Research and Therapy Program Wake Forest School of Medicine (WFSOM)  Winston Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6715-865X","authenticated-orcid":false,"given":"Gra\u00e7a","family":"Almeida\u2010Porada","sequence":"additional","affiliation":[{"name":"Wake Forest Institute for Regenerative Medicine Fetal Research and Therapy Program Wake Forest School of Medicine (WFSOM)  Winston Salem North Carolina 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