{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T07:40:31Z","timestamp":1792482031701,"version":"4.5.0"},"reference-count":108,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T00:00:00Z","timestamp":1783641600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T00:00:00Z","timestamp":1783641600000},"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 Disord Clin Pract"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Neurodegeneration with Brain Iron Accumulation (NBIA) is a heterogeneous group of heritable, mostly recessive, progressive neurodegenerative diseases characterized by iron deposition in the basal ganglia and brainstem. There are no solid global epidemiological data on prevalence and incidence of NBIA subtypes, but registry data and expert opinion suggest PKAN, BPAN, PLAN, and MPAN are the most common subtypes. NBIA disorders present with a wide spectrum of clinical symptoms, including movement disorders (dystonia, parkinsonism, chorea), pyramidal involvement (eg, spasticity), speech and cognitive deficits, motor and cognitive slowing, and ocular abnormalities. Treatment remains symptomatic, though several new drugs are in development.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives and methods<\/jats:title>\n                    <jats:p>Following our review published in 2021, this article provides an updated summary of recent developments. We discuss the rationale of new compounds, summarize clinical trials or\u2014in their absence\u2014preclinical studies for NBIA subtypes. The article is divided into two sections: one section on general approaches based on the shared feature of increased iron in the brain; and the second section on tailor\u2010made, mechanistic treatments for the various NBIA subtypes targeting the specific molecular and cellular pathways of the affected enzyme including gene therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results and conclusions<\/jats:title>\n                    <jats:p>In summary, randomized controlled trials in NBIA have not yet demonstrated substantial benefit, neither for iron removal, in general, which appears to be clinically ineffective in most subtypes, except aceruloplasminemia; nor for subtype\u2010specific approaches. Several ongoing studies are exploring more dedicated compounds in this exciting field.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mdc3.70736","type":"journal-article","created":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T10:31:10Z","timestamp":1783679470000},"page":"2405-2418","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Precision Medicine in Neurodegeneration with Brain Iron Accumulation (\n                    <scp>NBIA<\/scp>\n                    ) Disorders: An Update on Emerging Treatments"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7283-1995","authenticated-orcid":false,"given":"Susanne A.","family":"Schneider","sequence":"first","affiliation":[{"name":"Neuroimaging Center Medical Hospital of the Johannes Gutenberg University Mainz  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0532-3370","authenticated-orcid":false,"given":"Divyani","family":"Garg","sequence":"additional","affiliation":[{"name":"Department of Neurology All India Institute of Medical Sciences  New Delhi India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vassilena","family":"Iankova","sequence":"additional","affiliation":[{"name":"Department of Neurology M\u00fcnchen Klinik Harlaching  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Klopstock","sequence":"additional","affiliation":[{"name":"Friedrich Baur Institute, Department of Neurology LMU University Hospital, Ludwig\u2010Maximilians\u2010University Munich  Munich Germany"},{"name":"Munich Cluster for Systems Neurology (SyNergy)  Munich Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE)  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,7,10]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.3390\/ph12010027"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2021.629414"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2023.1098454"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtemb.2021.126779"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awaf398"},{"key":"e_1_2_10_7_1","unstructured":"FDA.https:\/\/www.fda.gov\/vaccines-blood-biologics\/cellular-gene-therapy-products\/what-gene-therapy[online]. 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