{"id":3419,"date":"2026-09-02T16:25:29","date_gmt":"2026-09-02T16:25:29","guid":{"rendered":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/?p=3419"},"modified":"2026-09-02T16:25:29","modified_gmt":"2026-09-02T16:25:29","slug":"new-eu-approved-medicine-for-rett-syndrome-offers-hope-for-children-and-families","status":"publish","type":"post","link":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/new-eu-approved-medicine-for-rett-syndrome-offers-hope-for-children-and-families\/","title":{"rendered":"New EU-Approved Medicine for Rett Syndrome Offers Hope for Children and Families"},"content":{"rendered":"

A new medication called Daybu (trofinetide) has been approved in Europe for children aged five and older who have Rett syndrome, a rare genetic disorder that affects brain development. Developed by Acadia Pharmaceuticals, Daybu is the first treatment in the European Union specifically for the neurobehavioral symptoms\u2014such as difficulty with movement, communication, and behavior\u2014caused by Rett syndrome. The active ingredient in Daybu is a molecule similar to a hormone<\/a> called IGF-1, which plays a key role in the growth and function of the nervous system. People with Rett syndrome often have low levels of this hormone. The European Commission\u2019s approval comes after the medication was already authorized for use in the United States and Canada. Dr. Nadia Bahi, a pediatric neurologist at Necker-Enfants Malades University Hospital in Paris, explained that Rett syndrome places a heavy burden on patients, their families, and caregivers, and that treatment options have been very limited until now. This approval is based on a large clinical study called the LAVENDER trial, which showed that Daybu significantly improved core symptoms of Rett syndrome, such as better communication, reduced repetitive movements, and improved ability to perform daily tasks. Catherine Owen Adams, the CEO of Acadia Pharmaceuticals, called the approval a major step forward for the Rett syndrome community in Europe. She emphasized that this medication addresses a long-standing unmet need for patients and families who have struggled with the challenges of this condition. In related news, Jazz Pharmaceuticals recently agreed to acquire Actio Biosciences for up to $1.32 billion. This move aims to advance a potential new treatment for a rare form of epilepsy linked to the KCNT1 gene, which also causes significant developmental and neurological<\/a> problems. If successful, this new therapy could provide another important option for patients with rare neurological disorders.<\/p>\n","protected":false},"excerpt":{"rendered":"

A new medication called Daybu (trofinetide) has been approved in Europe for children aged five and older who have Rett […]<\/p>\n","protected":false},"author":0,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[543],"tags":[],"class_list":["post-3419","post","type-post","status-publish","format-standard","hentry","category-pharmaceutical-news"],"_links":{"self":[{"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/posts\/3419","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/types\/post"}],"replies":[{"embeddable":true,"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/comments?post=3419"}],"version-history":[{"count":1,"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/posts\/3419\/revisions"}],"predecessor-version":[{"id":3425,"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/posts\/3419\/revisions\/3425"}],"wp:attachment":[{"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/media?parent=3419"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/categories?post=3419"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/allesleypharmacy.co.uk\/pharmacy\/wp-json\/wp\/v2\/tags?post=3419"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}