{"id":21964,"date":"2023-04-03T17:19:24","date_gmt":"2023-04-03T11:49:24","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=21964"},"modified":"2023-05-22T12:29:38","modified_gmt":"2023-05-22T06:59:38","slug":"daybue-for-rett-syndrome-treatment","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment","title":{"rendered":"DAYBUE: First and Only Approved Drug for Rett Syndrome Treatment"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_76 counter-hierarchy ez-toc-counter ez-toc-white ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-6a1cdba117cf2\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-6a1cdba117cf2\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#Rett_Syndrome_Treatment_Landscape\" >Rett Syndrome Treatment Landscape<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#Approval_of_DAYBUE_A_Long-awaited_Hope_for_Rett_Syndrome_Patients\" >Approval of DAYBUE: A Long-awaited Hope for Rett Syndrome Patients<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#Competitors_of_DAYBUE\" >Competitors of DAYBUE<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#Recent_Developments_in_the_Rett_Syndrome_Treatment_Space\" >Recent Developments in the Rett Syndrome Treatment Space<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#Promising_Future_of_Rett_Syndrome_Treatment_Market\" >Promising Future of Rett Syndrome Treatment Market<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment\/#FAQs\" >FAQs<\/a><\/li><\/ul><\/nav><\/div>\n\n<p>Rett syndrome is a rare progressive neurodevelopmental disorder primarily affecting women. Males are only occasionally affected. Infants with Rett syndrome typically develop normally for the first 7 to 18 months of life. The <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-epidemiology-forecast\">Rett syndrome prevalence<\/a> in the United States is estimated to be <strong>1 in 10K <\/strong>girls by the age of 12. Cases of Rett syndrome can go undiagnosed or be misdiagnosed, making it difficult to determine the disorder\u2019s true prevalence in the general population. Following Down syndrome, Rett syndrome is the second most common cause of severe intellectual disability.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-rett-syndrome-treatment-landscape\"><span class=\"ez-toc-section\" id=\"Rett_Syndrome_Treatment_Landscape\"><\/span><strong>Rett Syndrome Treatment Landscape<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>The treatment of Rett syndrome focuses on the specific symptoms that each person experiences. Treatment may necessitate the collaborative efforts of a team of specialists. Pediatricians, pediatric neurologists, gastroenterologists, speech therapists, psychiatrists, nutritionists, and other healthcare professionals may be required to plan an affected child\u2019s treatment systematically and comprehensively. Affected individuals and their families may benefit from genetic counseling. Individuals with Rett syndrome may benefit from a wide range of treatment options. The <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-market\">Rett syndrome treatment<\/a> plan will need to be highly personalized. Early developmental intervention ensures that affected children achieve their full potential. Occupational, physical, and speech therapy will be beneficial to the majority of affected children. Various rehabilitative and behavioral therapy methods may be beneficial. Additional medical, social, and vocational services, such as special education, may be required. Psychosocial support for the entire family is also required.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Approval_of_DAYBUE_A_Long-awaited_Hope_for_Rett_Syndrome_Patients\"><\/span><strong>Approval of DAYBUE: A Long-awaited Hope for Rett Syndrome Patients<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p><strong>10 March 2023<\/strong> was a very big day for all the <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-epidemiology-forecast\">Rett syndrome patients<\/a> and especially for <strong>Acadia Pharmaceuticals and Neuren Pharmaceuticals,<\/strong> as <strong>DAYBUE <\/strong>became the first and only drug approved for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. The FDA set the Prescription Drug User Fee Act (PDUFA) action date of <strong>12 March 2023<\/strong> but instead delivered the good news the late evening of <strong>10 March<\/strong>.&nbsp;<\/p>\n\n\n\n<p><strong>DAYBUE (trofinetide)<\/strong> is a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor-1, glycine-proline-glutamate. Though the mechanism by which trofinetide exerts its therapeutic effects in patients with Rett syndrome is unknown, animal studies suggest that it increases dendrite branching and synaptic plasticity signals. <strong>In 2018, Acadia <\/strong>signed an exclusive license agreement with <strong>Neuren Pharmaceuticals Limited<\/strong> for the development and commercialization of <strong>DAYBUE (trofinetide)<\/strong> in North America for the treatment of Rett syndrome and other indications.<\/p>\n\n\n\n<p>The FDA approved <strong>DAYBUE <\/strong>based on findings from the pivotal <strong>Phase III LAVENDER study<\/strong>, which compared the efficacy and safety of trofinetide versus placebo in 187 female Rett syndrome patients aged 5 to 20 years. At week 12, treatment with DAYBUE showed statistically significant improvement compared to placebo on both co-primary efficacy endpoints, as measured by change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and Clinical Global Impression-Improvement (CGI-I) scale score (p=0.003). The Rett Syndrome Behavior Questionnaire (RSBQ) is a caregiver assessment that evaluates a variety of Rett syndrome symptoms, such as vocalizations, facial expressions, eye gaze, hand movements (or stereotypies), repetitive behaviors, breathing, night-time behaviors, and mood. The CGI-I is a global physician evaluation of whether a patient has improved or deteriorated. The most common side effects in the study were diarrhea (82%), followed by vomiting (29%).<\/p>\n\n\n\n<figure class=\"wp-block-image size-full is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03155816\/DAYBUE-Roadmap.png\" alt=\"DAYBUE Roadmap\" class=\"wp-image-21986\" width=\"575\" height=\"234\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03155816\/DAYBUE-Roadmap.png 766w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03155816\/DAYBUE-Roadmap-300x122.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03155816\/DAYBUE-Roadmap-150x61.png 150w\" sizes=\"(max-width: 575px) 100vw, 575px\" \/><\/figure>\n\n\n\n<p>Acadia has received a <strong>Rare Pediatric Disease Priority Review Voucher<\/strong> as a result of the FDA approval of DAYBUE, which can be used to obtain priority review for a subsequent application. DAYBUE is expected to be available in the United States by the end of April 2023.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Competitors_of_DAYBUE\"><\/span><strong>Competitors of DAYBUE<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Several other pharma companies are also developing <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-market\">therapies for Rett syndrome treatment<\/a>. One such therapy and a competitor of <strong>DAYBUE <\/strong>is <strong>ANAVEX2-73. Anavex Life Sciences\u2019 ANAVEX2-73<\/strong> is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. The FDA has granted the drug <strong>Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation<\/strong> for the treatment of Rett syndrome. Currently, it is in the late stage of development for Rett syndrome treatment. It is anticipated that after the launch, ANAVEX2-73 will give tough competition to DAYBUE.&nbsp;<\/p>\n\n\n\n<p>Recently, the company announced the enrollment of 92 Rett syndrome patients for the <strong>ANAVEX\u00ae2-73 (blarcamesine) EXCELLENCE Phase II\/III study<\/strong> in Rett syndrome patients aged 5 to 17 years (inclusive). This exceeds the original enrollment goal, and the Company expects to release topline results from this study in the second half of 2023. The completion of enrollment in the randomized, placebo-controlled EXCELLENCE Phase II\/III study ANAVEX\u00ae2-73-RS-003 for the treatment of pediatric patients with Rett syndrome was preceded by the completion of both placebo-controlled <strong>Phase II U.S. (ANAVEX\u00ae2-73-RS-001)<\/strong> and <strong>Phase III AVATAR (ANAVEX\u00ae2-73-RS-002)<\/strong> studies in adult patients with Rett syndrome.&nbsp;<\/p>\n\n\n\n<p>Apart from ANAVEX2-73, the other therapies in the <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-pipeline-insight\">Rett syndrome pipeline<\/a> include <strong>Vatiquinone (PTC Therapeutics), Fingolimod (Novartis), Triheptanoin (Ultragenyx Pharmaceutical), Cannabidivarin (GW Pharmaceuticals), STRX 230 (StrideBio), AMO 04 (AMO Pharma), Pridopidine (Prilenia Therapeutics), NLX 101 (Neurolixis), BHV 5000 (Biohaven Pharmaceuticals)<\/strong>. The expected entry of these therapies will threaten DAYBUE\u2019s market position.&nbsp;<\/p>\n\n\n\n<figure class=\"wp-block-image size-full is-resized\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091936\/Emerging-Therapies-for-Rett-Syndrome-Treatment.png\" alt=\"Emerging Therapies for Rett Syndrome Treatment\" class=\"wp-image-21971\" width=\"636\" height=\"328\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091936\/Emerging-Therapies-for-Rett-Syndrome-Treatment.png 848w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091936\/Emerging-Therapies-for-Rett-Syndrome-Treatment-300x155.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091936\/Emerging-Therapies-for-Rett-Syndrome-Treatment-150x77.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091936\/Emerging-Therapies-for-Rett-Syndrome-Treatment-768x396.png 768w\" sizes=\"(max-width: 636px) 100vw, 636px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Recent_Developments_in_the_Rett_Syndrome_Treatment_Space\"><\/span><strong>Recent Developments in the Rett Syndrome Treatment Space<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>In <strong>March 2023, Neuren Pharmaceuticals<\/strong>, based in Melbourne, announced that its North American partner Acadia Pharmaceuticals had received US Food and Drug Administration (FDA) approval for Neuren&#8217;s lead candidate Daybue (trofinetide) to treat Rett syndrome in adults and children.<\/li>\n\n\n\n<li>In <strong>February 2023, Unravel Biosciences, Inc.<\/strong>, a therapeutics company that advances drugs for complex diseases by leveraging a machine-learned model of human health, announced the successful completion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) for its RVL001 program in Rett Syndrome.<\/li>\n\n\n\n<li>In <strong>February 2023, PharmaTher Holdings Ltd.<\/strong>, a leader in specialty ketamine pharmaceuticals, announced that the United States Food and Drug Administration had designated KETARX (racemic ketamine) as an orphan drug for Rett Syndrome treatment, a rare genetic neurological disorder.<\/li>\n\n\n\n<li>In <strong>February 2023, Anavex Life Sciences Corp.<\/strong> announced the enrollment of 92 Rett syndrome patients in the ANAVEX\u00ae2-73 (blarcamesine) EXCELLENCE Phase II\/III study in Rett syndrome patients aged 5 to 17 years (inclusive).<\/li>\n\n\n\n<li>In <strong>January 2023, Neurogene Inc.<\/strong>, a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced that the FDA has approved the Company\u2019s Investigational New Drug (IND) application for NGN-401 for Rett syndrome treatment.<\/li>\n\n\n\n<li>In <strong>October 2022, Taysha Gene Therapies, Inc. and Astellas Pharma Inc.<\/strong> announced a strategic investment to support Taysha\u2019s adeno-associated virus (AAV) gene therapy development programs for Rett syndrome treatment and GAN.<\/li>\n\n\n\n<li>In <strong>September 2022, Anavex<\/strong> announced that USPTO issued US Patent No. 11,446,275 to Anavex, with claims to treatment methods using its leading drug candidate, ANAVEX2-73 and its analogs, for ameliorating biochemical and functional abnormalities associated with methyl-CpG binding protein 2 defects (MeCP2 defects).<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Promising_Future_of_Rett_Syndrome_Treatment_Market\"><\/span><strong>Promising Future of Rett Syndrome Treatment Market<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>New therapeutic options are required to address the unmet need for effective and curative therapies for Rett syndrome treatment. This has created an opportunity for key players to develop novel therapies for Rett syndrome treatment. In addition, several academic institutions and pharmaceutical companies are currently conducting <a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-pipeline-insight\">Rett syndrome clinical trials<\/a> for the treatment of various Rett syndrome symptoms. Moreover, precision therapeutics, such as gene therapy, have the potential to change disease status rather than just modify symptoms because they target the underlying cause of the disorder. The recent success in this field is exemplified by the \u2018Zolgenzma\u2019 spinal muscular atrophy trial. Several pharmaceutical companies have committed to gene therapy programs for Rett syndrome treatment, with plans to begin clinical trials and change the Rett syndrome treatment space in the coming years.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"FAQs\"><\/span><strong>FAQs<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<div class=\"schema-faq wp-block-yoast-faq-block\"><div class=\"schema-faq-section\" id=\"faq-question-1680493362414\"><strong class=\"schema-faq-question\"><strong>1. What is Rett syndrome?<\/strong><\/strong> <p class=\"schema-faq-answer\">Rett syndrome is a rare neurodevelopmental disorder that primarily affects girls but can be seen in boys on rare occasions. Rett syndrome is not a degenerative disorder, but it does cause problems with brain function that affect cognitive, sensory, emotional, motor, and autonomic functions.<\/p> <\/div> <div class=\"schema-faq-section\" id=\"faq-question-1680493377843\"><strong class=\"schema-faq-question\"><strong>2. What causes Rett syndrome?<\/strong><\/strong> <p class=\"schema-faq-answer\">It is caused by X-chromosome mutations in the MECP2 gene. On the MECP2 gene, there are over 200 different mutations. Most of these mutations are found in eight distinct \u201chot spots.\u201d The majority of the time, Rett syndrome is misdiagnosed as autism, cerebral palsy, or global developmental delay. It affects all ethnic and racial groups.<\/p> <\/div> <div class=\"schema-faq-section\" id=\"faq-question-1680493390686\"><strong class=\"schema-faq-question\"><strong>3. What are the Rett syndrome symptoms?<\/strong><\/strong> <p class=\"schema-faq-answer\">Rett syndrome symptoms appear after an early period of apparently normal or near normal development until six to eighteen months of age when there is a slowing or stagnation of skills.<\/p> <\/div> <div class=\"schema-faq-section\" id=\"faq-question-1680493402483\"><strong class=\"schema-faq-question\"><strong>4. How is Rett syndrome treated?<\/strong><\/strong> <p class=\"schema-faq-answer\">The Rett syndrome treatment is symptomatic and supportive, focusing on symptom management, necessitating a multidisciplinary approach. Antiepileptic drugs can be used to help control seizures; selective serotonin reuptake inhibitors (SSRIs), a type of antidepressant, can be used to treat anxiety; proton pump inhibitors (PPIs) or H2 blockers can help reduce reflux; Constipation can be treated with laxatives.<\/p> <\/div> <\/div>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><a href=\"https:\/\/www.delveinsight.com\/report-store\/rett-syndrome-market\"><img decoding=\"async\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-1024x256.png\" alt=\"Rett Syndrome Market Outlook \" class=\"wp-image-21968\" width=\"768\" height=\"192\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-1024x256.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-300x75.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-150x38.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-768x192.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-1536x384.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook-1568x392.png 1568w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091830\/Rett-Syndrome-Market-Outlook.png 1584w\" sizes=\"(max-width: 768px) 100vw, 768px\" \/><\/a><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>Rett syndrome is a rare progressive neurodevelopmental disorder primarily affecting women. Males are only occasionally affected. Infants with Rett syndrome typically develop normally for the first 7 to 18 months of life. The Rett syndrome prevalence in the United States is estimated to be 1 in 10K girls by the age of 12. Cases of [&hellip;]<\/p>\n","protected":false},"author":14,"featured_media":21965,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":6,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[],"industry":[17225],"therapeutic_areas":[17238,17234],"class_list":["post-21964","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-articles","industry-pharmaceutical","therapeutic_areas-genetic-disorders","therapeutic_areas-rare-diseases"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v25.8 (Yoast SEO v25.8) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>DAYBUE: First &amp; Only Approved Drug for Rett Syndrome 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\/>\n\t<meta name=\"twitter:data1\" content=\"Sandeep Joshi\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"8 minutes\" \/>\n<!-- \/ Yoast SEO Premium plugin. -->","yoast_head_json":{"title":"DAYBUE: First & Only Approved Drug for Rett Syndrome Treatment","description":"DAYBUE (trofinetide) by Acadia Pharmaceuticals & Neuren Pharmaceuticals became the first & only FDA-approved drug for Rett syndrome treatment.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment","og_locale":"en_US","og_type":"article","og_title":"DAYBUE: First & Only Approved Drug for Rett Syndrome Treatment","og_description":"DAYBUE (trofinetide) by Acadia Pharmaceuticals & Neuren Pharmaceuticals became the first & only FDA-approved drug for Rett syndrome 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Joshi"},"sameAs":["http:\/\/Delveinsight.com"]},{"@type":"Question","@id":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493362414","position":1,"url":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493362414","name":"1. What is Rett syndrome?","answerCount":1,"acceptedAnswer":{"@type":"Answer","text":"Rett syndrome is a rare neurodevelopmental disorder that primarily affects girls but can be seen in boys on rare occasions. Rett syndrome is not a degenerative disorder, but it does cause problems with brain function that affect cognitive, sensory, emotional, motor, and autonomic functions.","inLanguage":"en-US"},"inLanguage":"en-US"},{"@type":"Question","@id":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493377843","position":2,"url":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493377843","name":"2. What causes Rett syndrome?","answerCount":1,"acceptedAnswer":{"@type":"Answer","text":"It is caused by X-chromosome mutations in the MECP2 gene. On the MECP2 gene, there are over 200 different mutations. Most of these mutations are found in eight distinct \u201chot spots.\u201d The majority of the time, Rett syndrome is misdiagnosed as autism, cerebral palsy, or global developmental delay. It affects all ethnic and racial groups.","inLanguage":"en-US"},"inLanguage":"en-US"},{"@type":"Question","@id":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493390686","position":3,"url":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493390686","name":"3. What are the Rett syndrome symptoms?","answerCount":1,"acceptedAnswer":{"@type":"Answer","text":"Rett syndrome symptoms appear after an early period of apparently normal or near normal development until six to eighteen months of age when there is a slowing or stagnation of skills.","inLanguage":"en-US"},"inLanguage":"en-US"},{"@type":"Question","@id":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493402483","position":4,"url":"https:\/\/www.delveinsight.com\/blog\/daybue-for-rett-syndrome-treatment#faq-question-1680493402483","name":"4. How is Rett syndrome treated?","answerCount":1,"acceptedAnswer":{"@type":"Answer","text":"The Rett syndrome treatment is symptomatic and supportive, focusing on symptom management, necessitating a multidisciplinary approach. Antiepileptic drugs can be used to help control seizures; selective serotonin reuptake inhibitors (SSRIs), a type of antidepressant, can be used to treat anxiety; proton pump inhibitors (PPIs) or H2 blockers can help reduce reflux; Constipation can be treated with laxatives.","inLanguage":"en-US"},"inLanguage":"en-US"}]}},"author_meta":{"display_name":"Sandeep Joshi","author_link":"https:\/\/www.delveinsight.com\/blog\/author\/sjoshidelveinsight-com"},"featured_img":"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2023\/04\/03091626\/daybue-for-rett-syndrome-treatment-300x187.png","coauthors":[],"tax_additional":{"categories":{"linked":["<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Articles<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">Articles<\/span>"]}},"comment_count":"0","relative_dates":{"created":"Posted 3 years ago","modified":"Updated 3 years ago"},"absolute_dates":{"created":"Posted on Apr 3, 2023","modified":"Updated on May 22, 2023"},"absolute_dates_time":{"created":"Posted on Apr 3, 2023 5:19 pm","modified":"Updated on May 22, 2023 12:29 pm"},"featured_img_caption":"daybue-for-rett-syndrome-treatment","series_order":"","_links":{"self":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/21964","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/users\/14"}],"replies":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/comments?post=21964"}],"version-history":[{"count":0,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/21964\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media\/21965"}],"wp:attachment":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media?parent=21964"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/categories?post=21964"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/tags?post=21964"},{"taxonomy":"industry","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/industry?post=21964"},{"taxonomy":"therapeutic_areas","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/therapeutic_areas?post=21964"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}