{"id":5483,"date":"2026-09-18T09:53:02","date_gmt":"2026-09-18T07:53:02","guid":{"rendered":"https:\/\/encore-expertisecentrum.nl\/?p=5483"},"modified":"2026-09-18T09:53:02","modified_gmt":"2026-09-18T07:53:02","slug":"nieuws-over-de-trials-met-anti-sense-oligonucleotiden-aso-bij-mensen-met-angelman-syndroom","status":"publish","type":"post","link":"https:\/\/encore-expertisecentrum.nl\/en\/nieuws-over-de-trials-met-anti-sense-oligonucleotiden-aso-bij-mensen-met-angelman-syndroom\/","title":{"rendered":"News about trials with antisense oligonucleotides (ASO) in people with Angelman syndrome"},"content":{"rendered":"<p><strong>We would like to provide a brief update on the three Phase 3 trials involving an ASO from the pharmaceutical industry.<\/strong><\/p>\n<p><strong>Ultragenyx<\/strong> investigated its drug GTX-102 (apazunersen) <em>in the Aspire study. <\/em>An analysis of the primary outcome measures was conducted very recently; these showed no significant improvement. Consequently, the study yielded negative results and is being discontinued.<\/p>\n<p>At ENCORE, we had decided at an earlier stage not to participate in this study.<\/p>\n<p><strong>Ionis<\/strong> is currently conducting the <em>Reveal and Champion<\/em> studies on ION582 (obudanersen); all participants have now started, and we eagerly await the results once they have completed their follow-up assessments.<\/p>\n<p>ENCORE would have liked to participate in this study, but the manufacturer's submission to the ethics committee (CCMO) in the Netherlands was rejected. Approval was granted in other countries.<\/p>\n<p><strong>Oak Hill Bio<\/strong> is in the start-up phase of the <em>Beacon <\/em>study on Rugonersen; this study has been submitted for ethical approval. We expect to be able to participate in it through ENCORE.<\/p>","protected":false},"excerpt":{"rendered":"<p>Graag geven we een korte update over de drie fase 3 onderzoeken met een ASO vanuit de farmaceutische industrie. Ultragenyx heeft in de Aspire studie onderzoek gedaan naar hun middel GTX-102 (apazunersen). Zeer recent is de analyse gedaan van de primaire uitkomstmaten. Deze bleken geen significante verbetering te tonen. Daarmee is de studie negatief ge\u00ebindigd [&hellip;]<\/p>\n","protected":false},"author":5,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":[],"categories":[3],"tags":[],"_links":{"self":[{"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/posts\/5483"}],"collection":[{"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/users\/5"}],"replies":[{"embeddable":true,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/comments?post=5483"}],"version-history":[{"count":1,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/posts\/5483\/revisions"}],"predecessor-version":[{"id":5484,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/posts\/5483\/revisions\/5484"}],"wp:attachment":[{"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/media?parent=5483"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/categories?post=5483"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/encore-expertisecentrum.nl\/en\/wp-json\/wp\/v2\/tags?post=5483"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}