{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T20:29:32Z","timestamp":1790713772280,"version":"4.1.0"},"reference-count":21,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2017,8,13]],"date-time":"2017-08-13T00:00:00Z","timestamp":1502582400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2017,8,13]],"date-time":"2017-08-13T00:00:00Z","timestamp":1502582400000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100004329","name":"Genzyme","doi-asserted-by":"publisher","award":["GZ-2015-11365"],"award-info":[{"award-number":["GZ-2015-11365"]}],"id":[{"id":"10.13039\/100004329","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004329","name":"Genzyme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004329","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Journal of Genetic Counseling"],"published-print":{"date-parts":[[2018,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Type 1 Gaucher disease (GD) is the most common lysosomal storage disorder. Previously, treatment for GD was limited to intravenous enzyme replacement therapies (ERTs). More recently, oral substrate reduction therapies (SRTs) were approved for treatment of GD. Although both therapies alleviate disease symptoms, attitudes toward SRTs and patient perceptions of health while using SRT have not been well established. Electronic surveys were administered to adults with GD and asked about treatment history, attitudes toward SRTs, and perception of health while using SRTs as compared to ERTs, if applicable to the participant. ERT users that were offered treatment with SRTs cited potential side effects, wanting more research on SRTs, and satisfaction with their current treatment regimen as reasons for declining SRTs. SRT users expressed convenience and less invasiveness as reasons for choosing SRTs. Additionally, those using SRTs most often perceived their health to be similar to when they previously used ERT. Participant responses illustrate that attitudes toward SRTs can be variable and that one particular treatment may not be ideal for all patients with GD depending on individual perceptions of factors such as convenience, invasiveness, or side effects. Thus, individuals with GD should be counseled adequately by healthcare providers about both ERTs and SRTs for treatment of GD now that SRTs are clinically available.<\/jats:p>","DOI":"10.1007\/s10897-017-0137-0","type":"journal-article","created":{"date-parts":[[2017,8,12]],"date-time":"2017-08-12T22:13:15Z","timestamp":1502575995000},"page":"169-176","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies"],"prefix":"10.1002","volume":"27","author":[{"given":"Victoria F.","family":"Wagner","sequence":"first","affiliation":[{"name":"Genetic Counseling Program The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences Houston TX USA"},{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hope","family":"Northrup","sequence":"additional","affiliation":[{"name":"Genetic Counseling Program The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences Houston TX USA"},{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S. Shahrukh","family":"Hashmi","sequence":"additional","affiliation":[{"name":"Genetic Counseling Program The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences Houston TX USA"},{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanne M.","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Genetic Counseling Program The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences Houston TX USA"},{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary Kay","family":"Koenig","sequence":"additional","affiliation":[{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica M.","family":"Davis","sequence":"additional","affiliation":[{"name":"Genetic Counseling Program The University of Texas MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences Houston TX USA"},{"name":"Department of Pediatrics McGovern Medical School at The University of Texas Health Science Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,8,13]]},"reference":[{"key":"e_1_2_11_2_1","first-page":"1354","article-title":"Gaucher's disease","volume":"19","author":"Beutler E.","year":"1991","journal-title":"New England Journal of Medicine"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/0006-291X(65)90743-6"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S92985"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(00)02161-9"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(14)61841-9"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-02-075960"},{"key":"e_1_2_11_8_1","first-page":"3","article-title":"Overview: Gaucher disease","volume":"2","author":"Esplin J. 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