{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,19]],"date-time":"2026-08-19T17:46:46Z","timestamp":1787161606241,"version":"build-2736575974"},"reference-count":17,"publisher":"Becaris Publishing Limited","issue":"1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:p>\n                    <jats:bold>Aim:<\/jats:bold>\n                    Patients with Rett syndrome (RTT) experience gastrointestinal (GI) manifestations. This study aimed to describe the prevalence of GI manifestations and the associated medical costs in patients with RTT in the USA.\n                    <jats:bold>Patients &amp; Methods:<\/jats:bold>\n                    The study combined an insurance claims database analysis with a survey of 100 physicians experienced in RTT management.\n                    <jats:bold>Results:<\/jats:bold>\n                    GI manifestations affected 43.0% of 5940 patients, with increased prevalence in pediatric patients (45.6%) relative to\u00a0adult patients (40.2%). Annualized mean medical cost of managing GI manifestations was $4473. Only 5.9\u20138.2% of neurologists and pediatricians ranked GI symptom management among the five most important treatment goals.\n                    <jats:bold>Conclusion:<\/jats:bold>\n                    Patients with RTT experience a high burden of GI manifestations, which translate to considerable medical costs. Importantly, the prevalence of GI manifestations was likely underestimated in this study, as only those symptoms which resulted in a healthcare encounter were captured.\n                  <\/jats:p>","DOI":"10.57264\/cer-2023-0054","type":"journal-article","created":{"date-parts":[[2023,11,16]],"date-time":"2023-11-16T09:31:26Z","timestamp":1700127086000},"source":"Crossref","is-referenced-by-count":7,"title":["Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data"],"prefix":"10.57264","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1258-6649","authenticated-orcid":false,"given":"Damian M","family":"May","sequence":"first","affiliation":[{"name":"Acadia Pharmaceuticals Inc., San Diego, CA\u00a092130, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Neul","sequence":"additional","affiliation":[{"name":"Vanderbilt University Medical Center, Nashville, TN\u00a037232, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jes\u00fas Eric","family":"Pi\u00f1a-Garza","sequence":"additional","affiliation":[{"name":"Centennial Children's Hospital, Nashville, TN\u00a037203, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kal\u00e9","family":"Kponee-Shovein","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ambika","family":"Satija","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malena","family":"Mahendran","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Montr\u00e9al, H3B 0G7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathaniel","family":"Downes","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristy","family":"Sheng","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neema","family":"Lema","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andra","family":"Boca","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Lefebvre","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Montr\u00e9al, H3B 0G7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Victor","family":"Abler","sequence":"additional","affiliation":[{"name":"Acadia Pharmaceuticals Inc., San Diego, CA\u00a092130, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James M","family":"Youakim","sequence":"additional","affiliation":[{"name":"Acadia Pharmaceuticals Inc., San Diego, CA\u00a092130, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy Y","family":"Cheng","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"key":"e_1_3_8_3_2","unstructured":"National Institutes of Health (Nih). 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