{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T00:09:47Z","timestamp":1792454987825,"version":"4.5.0"},"reference-count":18,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2024,10,26]],"date-time":"2024-10-26T00:00:00Z","timestamp":1729900800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Huntington's Disease"],"published-print":{"date-parts":[[2024,11]]},"abstract":"<jats:p>In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences\u2019 SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.<\/jats:p>","DOI":"10.1177\/18796397241293955","type":"journal-article","created":{"date-parts":[[2024,12,20]],"date-time":"2024-12-20T04:49:17Z","timestamp":1734670157000},"page":"409-418","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":18,"title":["Huntington\u2019s Disease Clinical Trials Update: September 2024"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0679-0117","authenticated-orcid":false,"given":"Mena","family":"Farag","sequence":"first","affiliation":[{"name":"Huntington's Disease Centre, UCL Queen Square Institute of Neurology, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2716-2045","authenticated-orcid":false,"given":"Sarah J","family":"Tabrizi","sequence":"additional","affiliation":[{"name":"Huntington's Disease Centre, UCL Queen Square Institute of Neurology, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward J","family":"Wild","sequence":"additional","affiliation":[{"name":"Huntington's Disease Centre, UCL Queen Square Institute of Neurology, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,10,26]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-170262"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-189001"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-189003"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-190001"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-199003"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-200002"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-229002"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-229006"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-239001"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-240017"},{"key":"e_1_3_3_12_2","unstructured":"PTC Therapeutics. An extension study to evaluate the long-term safety and efficacy of PTC518 in participants with Huntington\u2019s disease https:\/\/clinicaltrials.gov\/study\/NCT06254482 (2024 accessed 14 August 2024)."},{"key":"e_1_3_3_13_2","unstructured":"PTC Therapeutics. A study to evaluate the safety and efficacy of PTC518 in participants with Huntington\u2019s disease https:\/\/clinicaltrials.gov\/study\/NCT05358717 (2024 accessed 14 August 2024)."},{"key":"e_1_3_3_14_2","unstructured":"PTC Therapeutics. Interim PIVOT-HD results demonstrate evidence of favorable CNS biomarker and clinical effects at Month 12 in Huntington\u2019s disease patients https:\/\/ir.ptcbio.com\/node\/16811\/pdf (2024 accessed 14 August 2024)."},{"key":"e_1_3_3_15_2","unstructured":"UniQure Biopharma B.V. Safety and Proof-of-Concept (POC) study with AMT-130 in adults with early manifest Huntington\u2019s disease."},{"key":"e_1_3_3_16_2","unstructured":"UniQure Biopharma B.V. Safety and efficacy of AMT-130 in European adults with early manifest Huntington\u2019s disease."},{"key":"e_1_3_3_17_2","unstructured":"UniQure Biopharma B.V. uniQure announces positive interim data update demonstrating slowing of disease progression in Phase I\/II Trials of AMT-130 for Huntington\u2019s disease https:\/\/uniqure.gcs-web.com\/news-releases\/news-release-details\/uniqure-announces-positive-interim-data-update-demonstrating (2024 accessed 14 August 2024)."},{"key":"e_1_3_3_18_2","unstructured":"Wave Life Sciences Ltd. Study of WVE-003 in patients with Huntington\u2019s disease https:\/\/clinicaltrials.gov\/study\/NCT05032196 (2024 accessed 14 August 2024)."},{"key":"e_1_3_3_19_2","unstructured":"Wave Life Sciences Ltd. Wave life sciences announces positive results from Phase 1b\/2a SELECT-HD Trial with first clinical demonstration of Allele-selective mutant Huntingtin lowering in Huntington\u2019s disease https:\/\/ir.wavelifesciences.com\/news-releases\/news-release-details\/wave-life-sciences-announces-positive-results-phase-1b2a-select (2024 accessed 14 August 2024)."}],"container-title":["Journal of Huntington's Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/18796397241293955","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/18796397241293955","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/18796397241293955","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T08:51:53Z","timestamp":1783414313000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/18796397241293955"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,10,26]]},"references-count":18,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,11]]}},"alternative-id":["10.1177\/18796397241293955"],"URL":"https:\/\/doi.org\/10.1177\/18796397241293955","relation":{},"ISSN":["1879-6397","1879-6400"],"issn-type":[{"value":"1879-6397","type":"print"},{"value":"1879-6400","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,10,26]]}}}