{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T22:32:56Z","timestamp":1788993176535,"version":"build-2803163510"},"reference-count":22,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Pediatr Drugs"],"published-print":{"date-parts":[[2025,1]]},"DOI":"10.1007\/s40272-024-00670-6","type":"journal-article","created":{"date-parts":[[2025,1,10]],"date-time":"2025-01-10T10:50:51Z","timestamp":1736506251000},"page":"111-118","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Ganaxolone: A Review in Epileptic Seizures Associated with Cyclin-Dependent Kinase-Like 5 Deficiency Disorder"],"prefix":"10.1007","volume":"27","author":[{"given":"Sheridan M.","family":"Hoy","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,1,10]]},"reference":[{"issue":"6","key":"670_CR1","doi-asserted-by":"publisher","first-page":"563","DOI":"10.1016\/S1474-4422(22)00035-7","volume":"21","author":"H Leonard","year":"2022","unstructured":"Leonard H, Downs J, Benke TA, et al. CDKL5 deficiency disorder: clinical features, diagnosis, and management. Lancet Neurol. 2022;21(6):563\u201376.","journal-title":"Lancet Neurol"},{"issue":"1","key":"670_CR2","doi-asserted-by":"publisher","first-page":"40","DOI":"10.1186\/s11689-021-09384-z","volume":"13","author":"HE Olson","year":"2021","unstructured":"Olson HE, Daniels CI, Haviland I, et al. Current neurologic treatment and emerging therapies in CDKL5 deficiency disorder. J Neurodev Disord. 2021;13(1):40.","journal-title":"J Neurodev Disord"},{"key":"670_CR3","unstructured":"US National Library of Medicine. CDKL5 deficiency disorder. 2022. https:\/\/medlineplus.gov\/genetics\/condition\/cdkl5-deficiency-disorder\/. Accessed 22 Oct 2024."},{"key":"670_CR4","unstructured":"National Organization for Rare Disorders. CDKL5 deficiency disorder. 2024. https:\/\/rarediseases.org\/rare-diseases\/cdkl5\/. Accessed 22 Oct 2024."},{"issue":"20","key":"670_CR5","doi-asserted-by":"publisher","first-page":"5098","DOI":"10.3390\/ijms20205098","volume":"20","author":"SD Kadam","year":"2019","unstructured":"Kadam SD, Sullivan BJ, Goyal A, et al. Rett syndrome and CDKL5 deficiency disorder: from bench to clinic. Int J Mol Sci. 2019;20(20):5098.","journal-title":"Int J Mol Sci"},{"issue":"5","key":"670_CR6","doi-asserted-by":"publisher","first-page":"491","DOI":"10.1111\/gbb.12292","volume":"15","author":"S Sivilia","year":"2016","unstructured":"Sivilia S, Mangano C, Beggiato S, et al. CDKL5 knockout leads to altered inhibitory transmission in the cerebellum of adult mice. Genes Brain Behav. 2016;15(5):491\u2013502.","journal-title":"Genes Brain Behav"},{"key":"670_CR7","unstructured":"Marinus Pharmaceuticals Emerald Limited. ZTALMY 50 mg\/mL oral suspension: EU prescribing information. 2023. https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/ztalmy. Accessed 22 Oct 2024."},{"key":"670_CR8","unstructured":"Marinus Pharmaceuticals Emerald Limited. ZTALMY 50 mg\/mL oral suspension: UK summary of product characteristics. 2024. https:\/\/products.mhra.gov.uk\/. Accessed 22 Oct 2024."},{"issue":"3","key":"670_CR9","doi-asserted-by":"publisher","first-page":"1284","DOI":"10.1016\/S0022-3565(24)36511-5","volume":"280","author":"RB Carter","year":"1997","unstructured":"Carter RB, Wood PL, Wieland S, et al. Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one), a selective, high-affinity, steroid modulator of the gamma-aminobutyric acid(A) receptor. J Pharmacol Exp Ther. 1997;280(3):1284\u201395.","journal-title":"J Pharmacol Exp Ther"},{"issue":"11","key":"670_CR10","doi-asserted-by":"publisher","first-page":"1317","DOI":"10.1080\/14737175.2021.1904895","volume":"21","author":"S Lattanzi","year":"2021","unstructured":"Lattanzi S, Riva A, Striano P. Ganaxolone treatment for epilepsy patients: from pharmacology to place in therapy. Expert Rev Neurother. 2021;21(11):1317\u201332.","journal-title":"Expert Rev Neurother"},{"issue":"5","key":"670_CR11","doi-asserted-by":"publisher","first-page":"394","DOI":"10.1016\/S1474-4422(22)00127-2","volume":"21","author":"D Gill","year":"2022","unstructured":"Gill D. A potential new treatment for CDKL5 deficiency disorder. Lancet Neurol. 2022;21(5):394\u20135.","journal-title":"Lancet Neurol"},{"issue":"8","key":"670_CR12","doi-asserted-by":"publisher","first-page":"933","DOI":"10.1007\/s40265-022-01724-0","volume":"82","author":"YN Lamb","year":"2022","unstructured":"Lamb YN. Ganaxolone: first approval. Drugs. 2022;82(8):933\u201340.","journal-title":"Drugs"},{"issue":"3","key":"670_CR13","doi-asserted-by":"publisher","first-page":"81","DOI":"10.1007\/s40267-022-00976-w","volume":"39","author":"SJ Keam","year":"2023","unstructured":"Keam SJ, Al-Salama ZT. Ganaxolone in seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: a profile of its use in the USA. Drugs Ther Perspect. 2023;39(3):81\u20138.","journal-title":"Drugs Ther Perspect."},{"issue":"5","key":"670_CR14","doi-asserted-by":"publisher","first-page":"417","DOI":"10.1016\/S1474-4422(22)00077-1","volume":"21","author":"EM Pestana-Knight","year":"2022","unstructured":"Pestana-Knight EM, Amin S, Bahi-Buisson N, et al. Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2022;21(5):417\u201327.","journal-title":"Lancet Neurol"},{"key":"670_CR15","unstructured":"Specchio N, Masuoka L, Aimetti A, et al. Long-term, durable seizure frequency reduction in individuals with CDKL5 deficiency disorder (CDD) treated with ganaxolone [abstract no. 3.283]. In: 72nd Annual Meeting of the American Epilepsy Society. 2018."},{"key":"670_CR16","unstructured":"US National Institutes of Health. ClinicalTrials.gov identifier NCT02358538. 2023. https:\/\/clinicaltrials.gov\/study\/NCT02358538. Accessed 22 Oct 2024."},{"key":"670_CR17","unstructured":"European Medicines Agency. ZTALMY (ganaxolone): EU public assessment report. 2023. https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/ztalmy. Accessed 22 Oct 2024."},{"issue":"1","key":"670_CR18","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1111\/epi.17826","volume":"65","author":"HE Olson","year":"2024","unstructured":"Olson HE, Amin S, Bahi-Buisson N, et al. Long-term treatment with ganaxolone for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: two-year open-label extension follow-up. Epilepsia. 2024;65(1):37\u201345.","journal-title":"Epilepsia"},{"key":"670_CR19","doi-asserted-by":"publisher","first-page":"140","DOI":"10.1016\/j.ejpn.2024.06.005","volume":"51","author":"J Downs","year":"2024","unstructured":"Downs J, Jacoby P, Specchio N, et al. Effects of ganaxolone on non-seizure outcomes in CDKL5 deficiency disorder: double-blind placebo-controlled randomized trial. Eur J Paediatr Neurol. 2024;51:140\u20136.","journal-title":"Eur J Paediatr Neurol"},{"issue":"6","key":"670_CR20","doi-asserted-by":"publisher","first-page":"591","DOI":"10.1007\/s40263-022-00921-5","volume":"36","author":"W Hong","year":"2022","unstructured":"Hong W, Haviland I, Pestana-Knight E, et al. CDKL5 deficiency disorder-related epilepsy: a review of current and emerging treatment. CNS Drugs. 2022;36(6):591\u2013604.","journal-title":"CNS Drugs"},{"issue":"874695","key":"670_CR21","first-page":"1","volume":"13","author":"S Amin","year":"2022","unstructured":"Amin S, Monaghan M, Aledo-Serrano A, et al. International consensus recommendations for the assessment and management of individuals with CDKL5 deficiency disorder. Front Neurol. 2022;13(874695):1\u201316.","journal-title":"Front Neurol"},{"issue":"3","key":"670_CR22","doi-asserted-by":"publisher","first-page":"832","DOI":"10.1002\/epi4.12914","volume":"9","author":"S Amin","year":"2024","unstructured":"Amin S, M\u00f8ller RS, Aledo-Serrano A, et al. Providing quality care for people with CDKL5 deficiency disorder: a European expert panel opinion on the patient journey. Epilepsia Open. 2024;9(3):832\u201349.","journal-title":"Epilepsia Open."}],"container-title":["Pediatric Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40272-024-00670-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40272-024-00670-6\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40272-024-00670-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,1,28]],"date-time":"2025-01-28T05:40:31Z","timestamp":1738042831000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40272-024-00670-6"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1]]},"references-count":22,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2025,1]]}},"alternative-id":["670"],"URL":"https:\/\/doi.org\/10.1007\/s40272-024-00670-6","relation":{},"ISSN":["1174-5878","1179-2019"],"issn-type":[{"value":"1174-5878","type":"print"},{"value":"1179-2019","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1]]},"assertion":[{"value":"6 November 2024","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 January 2025","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The preparation of this review was not supported by any external funding.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"Sheridan M. Hoy is a salaried employee of Adis International Ltd\/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Authorship and Conflict of interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability"}}]}}