{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T23:14:36Z","timestamp":1790810076031,"version":"4.1.0"},"reference-count":98,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T00:00:00Z","timestamp":1789084800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T00:00:00Z","timestamp":1789084800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["movementdisorders.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Movement Disorders"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Childhood\u2010onset movement disorders comprise a heterogeneous group of rare conditions with substantial unmet therapeutic needs. Recent advances in disease gene discovery, mechanistic modeling, and translational platforms have accelerated the development of targeted therapies and enabled innovative clinical trial designs for small patient populations. To review novel and emerging therapies for childhood\u2010onset movement disorders, with a focus on pharmacologic strategies, disease\u2010modifying approaches, and patient\u2010centered precision therapies. We surveyed the literature, major conference proceedings, and expert networks to identify therapies approved, in clinical development, or supported by compelling preclinical data between 2022 and 2025. We focused on small molecules and genetic therapies for conditions in which movement disorders represent a prominent clinical feature. Small molecules were categorized as repurposed or novel drugs, whereas genetic therapies included gene replacement, gene editing, and\n                    <jats:styled-content style=\"fixed-case\">RNA<\/jats:styled-content>\n                    \u2010based expression modulation. Drug repurposing approaches have shown promise in disorders related to the\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>GNAO1<\/jats:italic>\n                    <\/jats:styled-content>\n                    ,\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>ATP1A3<\/jats:italic>\n                    <\/jats:styled-content>\n                    ,\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>ATM<\/jats:italic>\n                      ,\n                    <\/jats:styled-content>\n                    and\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>ADCY5<\/jats:italic>\n                    <\/jats:styled-content>\n                    genes. Novel small molecules have advanced for Friedreich's ataxia and Tourette's syndrome. Gene replacement therapies have demonstrated clinical benefit in select neurotransmitter disorders, whereas gene editing strategies have entered preclinical development for\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>ATP1A3<\/jats:italic>\n                    <\/jats:styled-content>\n                    \u2010related disease. Antisense oligonucleotide therapies have yielded encouraging early results across several conditions with prominent movement disorder phenotypes, including\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>KIF1A<\/jats:italic>\n                    <\/jats:styled-content>\n                    \u2010related neurological disorder, Angelman syndrome,\n                    <jats:styled-content style=\"fixed-case\">\n                      <jats:italic>SCN2A<\/jats:italic>\n                    <\/jats:styled-content>\n                    \u2010related neurodevelopmental disorder, and ataxia\u2010telangiectasia. Precision\u2010based therapeutic strategies are rapidly reshaping the treatment landscape for childhood\u2010onset movement disorders. Continued progress will depend on rigorous phenotyping, careful ethical oversight, and deliberate efforts to promote equitable global access to emerging therapies. \u00a9 2026 International Parkinson and Movement Disorder Society.\n                  <\/jats:p>","DOI":"10.1002\/mds.70495","type":"journal-article","created":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T08:57:53Z","timestamp":1789117073000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Novel and Emerging Therapies for Childhood\u2010Onset Movement Disorders"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7171-1406","authenticated-orcid":false,"given":"Claudio M.","family":"de Gusmao","sequence":"first","affiliation":[{"name":"Division of Movement Disorders, Department of Neurology Massachusetts General Brigham Hospitals, Harvard University  Boston Massachusetts 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University of Calgary  Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9727-3459","authenticated-orcid":false,"given":"Emmanuel","family":"Roze","sequence":"additional","affiliation":[{"name":"Sorbonne University, Paris Brain Institute, INSERM, CNRS, Assistance Publique\u2014H\u00f4pitaux de Paris, Institute of Neurology, Salp\u00eatri\u00e8re Hospital  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,9,11]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2024.10.005"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/cells14080559"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1038\/S41467-023-44264-1"},{"key":"e_1_2_9_5_1","volume-title":"GNAO1\u2010Related Disorder","author":"Briere 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