{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T23:57:16Z","timestamp":1792799836604,"version":"4.5.0"},"reference-count":341,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2022,4,1]],"date-time":"2022-04-01T00:00:00Z","timestamp":1648771200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Aotearoa Foundation, Centre for Brain Research and University of Auckland","award":["3705579"],"award-info":[{"award-number":["3705579"]}],"id":[{"id":"https:\/\/ror.org\/03b94tp07","id-type":"ROR","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100001505","name":"Health Research Council of New Zealand","doi-asserted-by":"crossref","award":["3627373"],"award-info":[{"award-number":["3627373"]}],"id":[{"id":"10.13039\/501100001505","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100003189","name":"Freemasons New Zealand","doi-asserted-by":"crossref","award":["3719321"],"award-info":[{"award-number":["3719321"]}],"id":[{"id":"10.13039\/501100003189","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100020395","name":"Alzheimers New Zealand Charitable Trust","doi-asserted-by":"crossref","award":["370836"],"award-info":[{"award-number":["370836"]}],"id":[{"id":"10.13039\/501100020395","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100012744","name":"Oakley Mental Health Research Foundation","doi-asserted-by":"crossref","award":["3725204"],"award-info":[{"award-number":["3725204"]}],"id":[{"id":"10.13039\/100012744","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100020396","name":"Alzheimers New Zealand","doi-asserted-by":"crossref","award":["3718869"],"award-info":[{"award-number":["3718869"]}],"id":[{"id":"10.13039\/501100020396","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100001543","name":"Neurological Foundation of New Zealand","doi-asserted-by":"crossref","award":["848010"],"award-info":[{"award-number":["848010"]}],"id":[{"id":"10.13039\/501100001543","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100014191","name":"Brain Research New Zealand","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100014191","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["J Cent Nerv Syst Dis"],"published-print":{"date-parts":[[2022,4]]},"abstract":"<jats:p>Huntington\u2019s disease (HD) is an autosomal neurodegenerative disease that is characterized by an excessive number of CAG trinucleotide repeats within the huntingtin gene ( HTT). HD patients can present with a variety of symptoms including chorea, behavioural and psychiatric abnormalities and cognitive decline. Each patient has a unique combination of symptoms, and although these can be managed using a range of medications and non-drug treatments there is currently no cure for the disease. Current therapies prescribed for HD can be categorized by the symptom they treat. These categories include chorea medication, antipsychotic medication, antidepressants, mood stabilizing medication as well as non-drug therapies. Fortunately, there are also many new HD therapeutics currently undergoing clinical trials that target the disease at its origin; lowering the levels of mutant huntingtin protein (mHTT). Currently, much attention is being directed to antisense oligonucleotide (ASO) therapies, which bind to pre-RNA or mRNA and can alter protein expression via RNA degradation, blocking translation or splice modulation. Other potential therapies in clinical development include RNA interference (RNAi) therapies, RNA targeting small molecule therapies, stem cell therapies, antibody therapies, non-RNA targeting small molecule therapies and neuroinflammation targeted therapies. Potential therapies in pre-clinical development include Zinc-Finger Protein (ZFP) therapies, transcription activator-like effector nuclease (TALEN) therapies and clustered regularly interspaced short palindromic repeats (CRISPR)\/CRISPR-associated system (Cas) therapies. This comprehensive review aims to discuss the efficacy of current HD treatments and explore the clinical trial progress of emerging potential HD therapeutics.<\/jats:p>","DOI":"10.1177\/11795735221092517","type":"journal-article","created":{"date-parts":[[2022,5,21]],"date-time":"2022-05-21T08:16:47Z","timestamp":1653121007000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":113,"title":["Current and Possible Future Therapeutic Options for Huntington\u2019s Disease"],"prefix":"10.1177","volume":"14","author":[{"given":"Mackenzie W.","family":"Ferguson","sequence":"first","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Connor J.","family":"Kennedy","sequence":"additional","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thulani H.","family":"Palpagama","sequence":"additional","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry J.","family":"Waldvogel","sequence":"additional","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard L. M.","family":"Faull","sequence":"additional","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3801-4956","authenticated-orcid":false,"given":"Andrea","family":"Kwakowsky","sequence":"additional","affiliation":[{"name":"Centre for Brain Research, Department of Anatomy and Medical Imaging, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand"},{"name":"Pharmacology and Therapeutics, School of Medicine, Galway Neuroscience Centre, National University of Ireland Galway, Galway, Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,5,21]]},"reference":[{"key":"bibr1-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/B978-0-12-381328-2.00014-6"},{"key":"bibr2-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/306234a0"},{"key":"bibr3-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/B978-0-12-801893-4.00001-8"},{"key":"bibr4-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/ng0893-387"},{"key":"bibr5-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(93)90585-E"},{"key":"bibr6-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S0300-9084(02)01398-6"},{"key":"bibr7-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.8.4604"},{"key":"bibr8-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-160205"},{"key":"bibr9-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainresbull.2009.07.010"},{"key":"bibr10-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/1531-8249(199901)45:1<25::AID-ART6>3.0.CO;2-E"},{"key":"bibr11-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnmol.2019.00258"},{"key":"bibr12-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/j.1460-9568.2008.06310.x"},{"key":"bibr13-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(07)60111-1"},{"key":"bibr14-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-40"},{"key":"bibr15-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25075"},{"key":"bibr16-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2009.02.003"},{"issue":"3","key":"bibr17-11795735221092517","author":"Mestre T","year":"2009","journal-title":"Cochrane Database Syst Rev"},{"key":"bibr18-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s11940-013-0219-8"},{"key":"bibr19-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-013-0244-z"},{"key":"bibr20-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-016-0670-4"},{"key":"bibr21-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/7854_2013_238"},{"key":"bibr22-11795735221092517","first-page":"62","volume":"45","author":"Craufurd D","year":"2002","journal-title":"Oxf Monogr Med Genet"},{"key":"bibr23-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2013-307343"},{"key":"bibr24-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/jnp.17.4.496"},{"key":"bibr25-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2019.01.039"},{"key":"bibr26-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2020.1792880"},{"key":"bibr27-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-200002"},{"key":"bibr28-11795735221092517","volume-title":"A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292","author":"Hoffmann-La Roche"},{"key":"bibr29-11795735221092517","unstructured":"Roche provides update on tominersen programme in manifest Huntington\u2019s disease [press release]. Basel, Switzerland, 22 March 2021 2021."},{"key":"bibr30-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s11910-017-0711-8"},{"key":"bibr31-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(17)30280-6"},{"key":"bibr32-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2019.104569"},{"key":"bibr33-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/jcb.24432"},{"key":"bibr34-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-020-0389-4"},{"key":"bibr35-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22168363"},{"issue":"2","key":"bibr36-11795735221092517","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1016\/S0306-4522(98)00004-9","volume":"86","author":"Smith Y","year":"1998","journal-title":"Neuroscience"},{"key":"bibr37-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410310412"},{"key":"bibr38-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.870010102"},{"key":"bibr39-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.85.15.5733"},{"key":"bibr40-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.58.2.273"},{"key":"bibr41-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/nrd2784"},{"key":"bibr42-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1208\/aapsj080478"},{"key":"bibr43-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000198586.85250.13"},{"issue":"2","key":"bibr44-11795735221092517","first-page":"136","volume":"11","author":"Kieburtz K","year":"2001","journal-title":"Neurology"},{"key":"bibr45-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.48.2.358"},{"key":"bibr46-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/s40734-017-0051-5"},{"key":"bibr47-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2016.8655"},{"issue":"12","key":"bibr48-11795735221092517","first-page":"690","volume":"33","author":"Yero T","year":"2008","journal-title":"Pharma Therapeutic"},{"key":"bibr49-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/s40734-017-0051-5"},{"key":"bibr50-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000005817"},{"key":"bibr51-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-130071"},{"key":"bibr52-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(07)60111-1"},{"key":"bibr53-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1080\/14737175.2018.1403899"},{"key":"bibr54-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2165\/00003495-199753020-00007"},{"issue":"2","key":"bibr55-11795735221092517","first-page":"685","volume":"244","author":"Janssen P","year":"1988","journal-title":"J Pharmacol Exp Therapeut"},{"key":"bibr56-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318263c443"},{"key":"bibr57-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-016-0566-3"},{"key":"bibr58-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.59.5.694"},{"key":"bibr59-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/1531-8257(199903)14:2<326::AID-MDS1019>3.0.CO;2-Q"},{"key":"bibr60-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s004150170071"},{"issue":"2","key":"bibr61-11795735221092517","author":"Duggan L","year":"2005","journal-title":"Cochrane Database Syst Rev"},{"issue":"1","key":"bibr62-11795735221092517","first-page":"69","volume":"14","author":"Squitieri F","year":"2001","journal-title":"Cognit Behav Neurol"},{"key":"bibr63-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/appi.ajp.158.10.1719"},{"key":"bibr64-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/026988110201600402"},{"key":"bibr65-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/appi.ps.52.7.967"},{"key":"bibr66-11795735221092517","author":"Marder SR","year":"1994","journal-title":"Am J Psychiatr"},{"key":"bibr67-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/sj.npp.1300603"},{"issue":"1","key":"bibr68-11795735221092517","author":"Komossa K","year":"2011","journal-title":"Cochrane Database Syst Rev"},{"key":"bibr69-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1080\/10401230701844802"},{"key":"bibr70-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/appi.ajp.158.5.765"},{"key":"bibr71-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S0924-9338(01)00604-6"},{"key":"bibr72-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1097\/00004850-199403001-00004"},{"key":"bibr73-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2165\/00003088-199700321-00003"},{"key":"bibr74-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/0269881198012003021"},{"key":"bibr75-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.cellsig.2004.12.007"},{"key":"bibr76-11795735221092517","unstructured":"Chu A, Wadhwa R. Selective Serotonin Reuptake Inhibitors. In: StatPearls. Treasure Island (FL): StatPearls Publishing. Copyright \u00a9 2021, StatPearls Publishing LLC.; 2021."},{"issue":"5","key":"bibr77-11795735221092517","volume":"3","author":"Khushboo SB","year":"2017","journal-title":"J Applied Biotechnol Bioengineer"},{"key":"bibr78-11795735221092517","doi-asserted-by":"crossref","first-page":"20","DOI":"10.4088\/JCP.v64n0106","volume":"64","author":"Hirschfeld R","year":"2003","journal-title":"J Clin Psychiatr"},{"key":"bibr79-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/appi.ajp.2007.07030454"},{"issue":"1","key":"bibr80-11795735221092517","first-page":"4","volume":"25","author":"Garland EJ","year":"2016","journal-title":"J Canad Acad Child Adol Psychiat"},{"key":"bibr81-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25980"},{"key":"bibr82-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25750"},{"key":"bibr83-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s002130000575"},{"key":"bibr84-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2165\/00003495-198632060-00002"},{"key":"bibr85-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1176\/jnp.8.3.338"},{"issue":"3","key":"bibr86-11795735221092517","first-page":"216","volume":"15","author":"Patzold T","year":"2002","journal-title":"Cognit Behav Neurol"},{"key":"bibr87-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1097\/00005053-197502000-00006"},{"key":"bibr88-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1037\/1064-1297.13.1.72"},{"key":"bibr89-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3109\/09638230903469186"},{"key":"bibr90-11795735221092517","doi-asserted-by":"crossref","first-page":"28","DOI":"10.4088\/JCP.v65n0105","volume":"65","author":"Yatham LN","year":"2004","journal-title":"J Clin Psychiatr"},{"key":"bibr91-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/0269881111431751"},{"key":"bibr92-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/pcn.12186"},{"key":"bibr93-11795735221092517","first-page":"713","volume":"44","author":"Macdonald R","year":"1986","journal-title":"Adv Neurol"},{"key":"bibr94-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/archdischild-2012-302822"},{"key":"bibr95-11795735221092517","volume-title":"Pathophysiology of Disease: An Introduction to Clinical Medicine","author":"Hammer GD","year":"2014"},{"key":"bibr96-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.pharmthera.2006.07.005"},{"key":"bibr97-11795735221092517","doi-asserted-by":"publisher","DOI":"10.31887\/DCNS.2008.10.1\/hcrgrunze"},{"key":"bibr98-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1566-2772(02)00044-0"},{"key":"bibr99-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(10)70245-3"},{"key":"bibr100-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/npp.2011.128"},{"key":"bibr101-11795735221092517","doi-asserted-by":"publisher","DOI":"10.7150\/ijbs.9898"},{"key":"bibr102-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/0006-8993(93)91660-K"},{"key":"bibr103-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neulet.2006.08.036"},{"key":"bibr104-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.53.5.1000"},{"key":"bibr105-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1023\/A:1014814924965"},{"issue":"2","key":"bibr106-11795735221092517","first-page":"829","volume":"266","author":"Lang DG","year":"1993","journal-title":"J Pharmacol Exp Therapeut"},{"key":"bibr107-11795735221092517","doi-asserted-by":"publisher","DOI":"10.4103\/0378-6323.16782"},{"key":"bibr108-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/0888439002250448"},{"key":"bibr109-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.pjnns.2015.09.002"},{"key":"bibr110-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2217\/nmt.11.86"},{"key":"bibr111-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2522\/ptj.20070346"},{"key":"bibr112-11795735221092517","doi-asserted-by":"publisher","DOI":"10.5334\/tohm.381"},{"key":"bibr113-11795735221092517","volume-title":"Communication Device and Method for Deaf and Mute Persons","author":"Butnaru H","year":"2001"},{"issue":"2","key":"bibr114-11795735221092517","author":"Pennington L","year":"2004","journal-title":"Cochrane Database Syst Rev"},{"key":"bibr115-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.pjnns.2014.10.006"},{"issue":"710","key":"bibr116-11795735221092517","volume":"10","author":"Bachoud-L\u00e9vi A-C","year":"2019","journal-title":"Front Neurol"},{"issue":"32","key":"bibr117-11795735221092517","volume":"9","author":"Nagel SJ","year":"2015","journal-title":"Front Syst Neurosci"},{"key":"bibr118-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.jocn.2014.11.008"},{"key":"bibr119-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neulet.2006.07.036"},{"key":"bibr120-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci6030038"},{"key":"bibr121-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3171\/2014.2.JNS131722"},{"key":"bibr122-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3171\/2013.10.JNS131225"},{"key":"bibr123-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/7854_2013_238"},{"issue":"6","key":"bibr124-11795735221092517","first-page":"707","volume":"15","author":"Scoles DR","year":"2018","journal-title":"RNA Biol"},{"key":"bibr125-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-189001"},{"key":"bibr126-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21062146"},{"key":"bibr127-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2012.05.009"},{"key":"bibr128-11795735221092517","volume-title":"Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants with Early Manifest Huntington's Disease","author":"Ionis Pharmaceuticals I","year":"2019"},{"key":"bibr129-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1900907"},{"key":"bibr130-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9062004"},{"key":"bibr131-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-199003"},{"key":"bibr132-11795735221092517","unstructured":"Hoffmann-La Roche. A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) 2019 [updated June 9 20202; cited 2021 08\/01\/2021]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03342053."},{"key":"bibr133-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-190001"},{"key":"bibr134-11795735221092517","unstructured":"Hoffmann-La Roche. An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Patients Who Participated in Prior Roche and Genentech Sponsored Studies 2019 [updated December 10 2020; cited 2021 08\/01\/2021]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03842969."},{"key":"bibr135-11795735221092517","unstructured":"Hoffmann-La Roche. A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease 2019 [cited 2021 08\/01\/2021]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04000594."},{"issue":"16","key":"bibr136-11795735221092517","doi-asserted-by":"crossref","first-page":"P2.006","DOI":"10.1212\/WNL.88.16_supplement.P2.006","volume":"88","author":"Hersch S","year":"2017","journal-title":"Neurology"},{"key":"bibr137-11795735221092517","first-page":"3","volume":"9","author":"Kaemmerer WF","year":"2019","journal-title":"Degener Neurol Neuromuscul Dis"},{"key":"bibr138-11795735221092517","unstructured":"Wave Life Sciences Announces Topline Data and Addition of Higher Dose Cohort in Ongoing Phase 1b\/2a PRECISION-HD2 Trial in Huntington\u2019s Disease [press release]. Cambridge, Massachusetts: Wave Life Sciences, December 30 2019 2019."},{"key":"bibr139-11795735221092517","unstructured":"Wave Life Sciences Provides Update on Phase 1b\/2a PRECISION-HD Trials. [press release]. March 29 2021."},{"key":"bibr140-11795735221092517","unstructured":"Wave Life Sciences Reports Third Quarter 2020 Financial Results and Provides Business Update [press release]. Cambridge, Massachusetts: Wave Life Sciences, November 9 2020 2020."},{"issue":"15","key":"bibr141-11795735221092517","doi-asserted-by":"crossref","first-page":"4703","DOI":"10.1212\/WNL.94.15_supplement.4703","volume":"94","author":"Dale E","year":"2020","journal-title":"Neurology"},{"key":"bibr142-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0024308"},{"key":"bibr143-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0171127"},{"key":"bibr144-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1602\/neurorx.2.3.447"},{"key":"bibr145-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0049838"},{"key":"bibr146-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0107434"},{"key":"bibr147-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2014.153"},{"key":"bibr148-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aar3959"},{"key":"bibr149-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2013.0452"},{"key":"bibr150-11795735221092517","unstructured":"Triplet Therapeutics Presents Preclinical Data for TTX-3360, Announces Equity Pledge at the CHDI Foundation\u2019s 16th Annual HD Therapeutics Conference [press release]. April 27 2021."},{"key":"bibr151-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0801775105"},{"key":"bibr152-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/nature04791"},{"key":"bibr153-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/s40035-017-0101-9"},{"key":"bibr154-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2016.7"},{"key":"bibr155-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2017.71"},{"key":"bibr156-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.09.010"},{"issue":"6","key":"bibr157-11795735221092517","first-page":"1115","volume":"26","author":"Southwell AL","year":"2017","journal-title":"Hum Mol Genet"},{"issue":"1","key":"bibr158-11795735221092517","first-page":"36","volume":"48","author":"Caron NS","year":"2020","journal-title":"Nucleic Acids Res"},{"key":"bibr159-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.03.002"},{"key":"bibr160-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.06.021"},{"key":"bibr161-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci11020129"},{"key":"bibr162-11795735221092517","unstructured":"UniQure. Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington Disease 2019 [updated October 28 2020; cited 2021 09\/01\/2021]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04120493."},{"key":"bibr163-11795735221092517","unstructured":"uniQure Announces Completion of Enrollment in First Cohort of Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease [press release]. Amsterdam, The Netherlands, April 5, 2021 2021."},{"key":"bibr164-11795735221092517","unstructured":"UniQure Announces Positive Recommendation from Data Safety Monitoring Board of Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease [press release]. Lexington, Massachusetts; Amsterdam, The Netherlands, UniQure, February 8 2021 2021."},{"key":"bibr165-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2013.200"},{"key":"bibr166-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainres.2014.06.011"},{"key":"bibr167-11795735221092517","unstructured":"Voyager Therapeutics Announces Preclinical Data For Huntington\u2019s Disease And Amyotrophic Lateral Sclerosis Programs At The Congress Of The European Society Of Gene And Cell Therapy [press release]. Cambridge, Massachusetts, October 16 2018."},{"key":"bibr168-11795735221092517","unstructured":"Voyager Therapeutics Provides Regulatory Update On VY-HTT01 Program [press release]. Cambridge, Massachusetts: Voyager Therapeutics, October 13 2020."},{"key":"bibr169-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0501507102"},{"key":"bibr170-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.50"},{"key":"bibr171-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.219"},{"key":"bibr172-11795735221092517","unstructured":"Bhattacharyya A, ed. Identification and development of orally administered, CNS-penetrant small molecules that lower huntingtin protein levels by inducing a novel splicing event that alters the stability of huntingtin mRNA. CHDI HD Therapeutics Conference; 2019; Palm Springs, California."},{"key":"bibr173-11795735221092517","unstructured":"PTC Therapeutics Announces that PTC518 Has Entered into a Phase 1 Clinical Trial for the Huntington's Disease Program [press release]. South Plainfield, New Jersey2020."},{"key":"bibr174-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.0857-08.2008"},{"key":"bibr175-11795735221092517","unstructured":"Therapeutics PTC. PTC518 Phase 1 Huntington's Disease Program Update 2021 [cited 2022 January]. Available from: https:\/\/ir.ptcbio.com\/events\/event-details\/ptc518-phase-1-huntingtons-disease-program-update."},{"key":"bibr176-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-018-0868-8"},{"key":"bibr177-11795735221092517","unstructured":"Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington\u2019s disease (HD) [press release]. Basel, Switzerland, October 21 2020."},{"key":"bibr178-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2011.12.032"},{"key":"bibr179-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1005043"},{"key":"bibr180-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddy061"},{"key":"bibr181-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/1087057113510320"},{"key":"bibr182-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-019-53410-z"},{"key":"bibr183-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1021\/acschembio.7b00699"},{"key":"bibr184-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.biochi.2019.05.001"},{"key":"bibr185-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/1752-0509-4-29"},{"key":"bibr186-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1206506109"},{"key":"bibr187-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/s13024-016-0128-x"},{"key":"bibr188-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-019-0478-3"},{"key":"bibr189-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1602\/neurorx.2.3.465"},{"key":"bibr190-11795735221092517","doi-asserted-by":"publisher","DOI":"10.32607\/20758251-2014-6-3-19-40"},{"key":"bibr191-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3727\/096368916X690863"},{"key":"bibr192-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0095611"},{"key":"bibr193-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2018.00075"},{"key":"bibr194-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.stemcr.2017.01.022"},{"issue":"20","key":"bibr195-11795735221092517","first-page":"4566","volume":"25","author":"Shin JW","year":"2016","journal-title":"Hum Mol Genet"},{"key":"bibr196-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1172\/JCI92087"},{"key":"bibr197-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2019.07.009"},{"key":"bibr198-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2016.11.010"},{"key":"bibr199-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fncel.2020.00250"},{"key":"bibr200-11795735221092517","unstructured":"Azidus Brasil. Safety Evaluation of Cellavita HD Administered Intravenously in Participants With Huntington's Disease (SAVE-DH) 2016 [cited 2021 13 January]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02728115."},{"key":"bibr201-11795735221092517","unstructured":"Azidus Brasil. Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease (ADORE-DH) 2017. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03252535."},{"key":"bibr202-11795735221092517","unstructured":"Azidus Brasil. Clinical Extension Study for Safety and Efficacy Evaluation of Cellavita-HD Administration in Huntington's Patients. (ADORE-EXT) 2020 [cited 2021 13 January]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04219241."},{"key":"bibr203-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp.73.6.678"},{"key":"bibr204-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2012-302441"},{"key":"bibr205-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(00)03310-9"},{"key":"bibr206-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2013-306533"},{"key":"bibr207-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/ISRCTN52651778"},{"key":"bibr208-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1006\/exnr.1999.7239"},{"key":"bibr209-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25354"},{"key":"bibr210-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0000166"},{"key":"bibr211-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/j.1759-1961.2011.00018.x"},{"key":"bibr212-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(06)70381-7"},{"key":"bibr213-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/aws359"},{"key":"bibr214-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41536-019-0066-7"},{"key":"bibr215-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/ana.21788"},{"key":"bibr216-11795735221092517","unstructured":"Welter R. Autologous Stem\/Stromal Cells in Neurological Disorders and Disease (NDD) 2017 [cited 2021 20 February]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03297177."},{"key":"bibr217-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1590\/s1980-5764-2016dn1004006"},{"key":"bibr218-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2217\/rme.15.25"},{"key":"bibr219-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/scrt341"},{"key":"bibr220-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s12640-018-9884-4"},{"key":"bibr221-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2990.1999.00212.x"},{"key":"bibr222-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbr.2010.11.006"},{"key":"bibr223-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/scrt326"},{"key":"bibr224-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0101185"},{"key":"bibr225-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/0014-4886(89)90170-2"},{"key":"bibr226-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2020.558204"},{"key":"bibr227-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2017.00628"},{"key":"bibr228-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/1091581817740873"},{"key":"bibr229-11795735221092517","unstructured":"Keswani SC. Phase 2a Study of ANX005, a Humanized anti-C1q mAb, in Patients with Huntington disease. Huntington Study Group Annual Conference; October 30; Online2020."},{"key":"bibr230-11795735221092517","doi-asserted-by":"publisher","DOI":"10.14336\/AD.2019.0118"},{"key":"bibr231-11795735221092517","unstructured":"Annexon I. Single Dose Study of ANX005 in Healthy Volunteers 2017. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03010046."},{"key":"bibr232-11795735221092517","unstructured":"Annexon I. A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barr\u00e9 Syndrome (GBS) 2019. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04035135 https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04035135."},{"key":"bibr233-11795735221092517","unstructured":"Annexon I. An Open Label Study of ANX005 in Subjects With, or at Risk for, Manifest Huntington's Disease 2020 [cited 2021 26 January]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04514367."},{"key":"bibr234-11795735221092517","unstructured":"Annexon I. Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS) 2020. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04569435."},{"key":"bibr235-11795735221092517","unstructured":"Annexon I. Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ANX005 in Subjects With Warm Autoimmune Hemolytic Anemia (wAIHA) 2020 [cited 2021 28 January]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04691570."},{"key":"bibr236-11795735221092517","doi-asserted-by":"crossref","unstructured":"Islam Z, Papri N, Jahan I, Azad KAK, Kroon H, Humphriss E, et al. Inhibition of C1q, initiator of the classical complement cascade, by ANX005 for the treatment of Guillain-Barr\u00e9 Syndrome. Results from a Phase 1b study. 2020 American Academy of Neurology (AAN) Annual Meeting; 28 April; Toronto, Canada2020.","DOI":"10.1212\/WNL.94.15_supplement.763"},{"key":"bibr237-11795735221092517","unstructured":"Annexon I. Efficacy and Safety of ANX005 in Subjects With Guillain-Barr\u00e9 Syndrome 2021. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04701164."},{"key":"bibr238-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1158\/1535-7163.MCT-14-0924"},{"key":"bibr239-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2014.10.008"},{"key":"bibr240-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2015.01.002"},{"key":"bibr241-11795735221092517","unstructured":"Vaccinex I. A Study in Subjects With Late Prodromal and Early Manifest Huntington's Disease (HD) to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of Pepinemab (VX15\/2503) (SIGNAL) 2015. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02481674."},{"key":"bibr242-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-189003"},{"key":"bibr243-11795735221092517","unstructured":"Vaccinex, Inc. Announces Preliminary Data from the SIGNAL Clinical Trial (Investigational Drug VX15\/2503 as a Potential Treatment for Huntington\u2019s Disease) [press release]. Rochester, New York 2017."},{"key":"bibr244-11795735221092517","unstructured":"Top-Line Results of Phase 2 SIGNAL Study in Huntington\u2019s Disease Support Potential for Cognitive Benefit of Pepinemab [press release]. September 22 2020 2020."},{"key":"bibr245-11795735221092517","unstructured":"Vaccinex I. Learnings from the SIGNAL Phase 2 Study of Treatment with Pepinemab Antibody. Huntington Study Group 2020 Medical Conference; 30 October 2020; Virtual2020."},{"key":"bibr246-11795735221092517","unstructured":"University of Utah. Anti-SEMA4D Monoclonal Antibody VX15\/2503 With Nivolumab or Ipilimumab in Treating Patients With Stage III or IV Melanoma 2018. Available from: https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03425461?term=VX15&draw=5&rank=2."},{"key":"bibr247-11795735221092517","unstructured":"Vaccinex I. SEMA4D Blockade Safety and Brain Metabolic Activity in Alzheimer's Disease (AD) (SIGNAL-AD) 2020. Available from: https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT04381468."},{"key":"bibr248-11795735221092517","unstructured":"Emory University. VX15\/2503 and Immunotherapy in Resectable Pancreatic and Colorectal Cancer 2017. Available from: https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03373188 https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03373188."},{"key":"bibr249-11795735221092517","unstructured":"Emory University. VX15\/2503 in Combination With Ipilimumab or Nivolumab in Patients With Head and Neck Cancer 2018. Available from: https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03690986."},{"key":"bibr250-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/NXI.0000000000000367"},{"key":"bibr251-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-15-0431"},{"key":"bibr252-11795735221092517","unstructured":"Vaccinex I. VX15\/2503 in Combination With Avelumab in Advanced Non-small Cell Lung Cancer (CLASSICAL-Lung) 2017. Available from. https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03268057."},{"key":"bibr253-11795735221092517","volume-title":"Neurobiology of Huntington's Disease: Applications to Drug Discovery","author":"Khoshnan A","year":"2011"},{"key":"bibr254-11795735221092517","first-page":"7120753","volume":"2016","author":"Amaro IA","year":"2016","journal-title":"J Neurodegn Dis"},{"key":"bibr255-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/srep36631"},{"key":"bibr256-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.2747-08.2008"},{"key":"bibr257-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2004.11.003"},{"key":"bibr258-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0029199"},{"key":"bibr259-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41380-018-0021-9"},{"key":"bibr260-11795735221092517","unstructured":"Positive preclinical in vivo results with AFFiRiS\u2019 antibody mAB C6-17 to treat Huntington\u2019s disease to be presented at the 16th Annual Huntington's Disease Therapeutics Conference [press release]. April 27 2021."},{"key":"bibr261-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2020.104943"},{"key":"bibr262-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-170267"},{"key":"bibr263-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30391-0"},{"key":"bibr264-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddw238"},{"key":"bibr265-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/jcmm.12604"},{"key":"bibr266-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2016.10.006"},{"key":"bibr267-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1097\/WNF.0b013e3181ebb285"},{"key":"bibr268-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(11)70233-2"},{"key":"bibr269-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25362"},{"key":"bibr270-11795735221092517","unstructured":"Prilenia. A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Versus Placebo for Symptomatic Treatment in Patients With Huntington's Disease 2013. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02006472."},{"key":"bibr271-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-200440"},{"key":"bibr272-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182886965"},{"key":"bibr273-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-170241"},{"key":"bibr274-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-190393"},{"key":"bibr275-11795735221092517","unstructured":"Prilenia. A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease (Open PRIDE-HD) 2015. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02494778."},{"key":"bibr276-11795735221092517","unstructured":"Prilenia. PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD 2020. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04556656."},{"key":"bibr277-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-170262"},{"key":"bibr278-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/1756285613499424"},{"key":"bibr279-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-012-1009-1"},{"key":"bibr280-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2013-306132"},{"key":"bibr281-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s12035-018-1393-1"},{"key":"bibr282-11795735221092517","unstructured":"Teva Pharmaceutical Industries. A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod (LEGATO-HD) 2014 [updated May 4 2020; cited 2021 17 February]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02215616."},{"issue":"15","key":"bibr283-11795735221092517","doi-asserted-by":"crossref","first-page":"S16.007","DOI":"10.1212\/WNL.92.15_supplement.S16.007","volume":"92","author":"Reilmann R","year":"2019","journal-title":"Neurology"},{"key":"bibr284-11795735221092517","volume-title":"International Congress of Parkinson\u2019s Disease and Movement disorders","author":"Leavitt BR","year":"2019"},{"key":"bibr285-11795735221092517","volume-title":"International Congress of Parkinson\u2019s Disease and Movement disorders","author":"Reilmann R","year":"2019"},{"key":"bibr286-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1186\/1742-2094-8-79"},{"key":"bibr287-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JAD-150277"},{"issue":"8","key":"bibr288-11795735221092517","first-page":"1070","volume":"2","author":"Haddad JJ","year":"2001","journal-title":"Curr Opin Invest Drugs"},{"key":"bibr289-11795735221092517","unstructured":"EIP Pharma Inc. Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease 2019 [cited 2021 18 February]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03980938."},{"key":"bibr290-11795735221092517","unstructured":"EIP Pharma Announces Positive Phase 2 Results for Neflamapimod in Mild-to-Moderate Dementia with Lewy bodies (DLB) [press release]. Boston, Massachusetts, 6 October 2020 2020."},{"key":"bibr291-11795735221092517","unstructured":"EIP Pharma Announces Clinical Trial Results of REVERSE-SD, a Phase 2b Study of Neflamapimod in Early-stage Alzheimer\u2019s Disease [press release]. Boston, Massachusetts, November 7 2019."},{"key":"bibr292-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JPD-160867"},{"key":"bibr293-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddt192"},{"key":"bibr294-11795735221092517","doi-asserted-by":"publisher","DOI":"10.4172\/2155-9899.1000259"},{"key":"bibr295-11795735221092517","unstructured":"Anderson KE. Nilotinib in Huntington's Disease (Tasigna HD) 2018 [cited 2021 20 February ]. Available from. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03764215."},{"key":"bibr296-11795735221092517","unstructured":"Pfizer. Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease 2014 [cited 2021 18 February]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02197130."},{"key":"bibr297-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2016.10.064"},{"key":"bibr298-11795735221092517","unstructured":"Pfizer. Product Pipeline for New Pharmaceutical Drugs 2021 [updated February 2 2021; cited 2021 18 February]. Available from. https:\/\/www.pfizer.com\/science\/drug-product-pipeline."},{"key":"bibr299-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/bph.13438"},{"key":"bibr300-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.freeradbiomed.2013.04.016"},{"key":"bibr301-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9113682"},{"key":"bibr302-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2147\/NDT.S111083"},{"key":"bibr303-11795735221092517","unstructured":"Sage Therapeutics Announces Planned Progression of SAGE-718 to Phase 2 in Huntington's Disease and Presentations at the 2019 Annual Meeting of the American College of Neuropsychopharmacology (ACNP) [press release]. December 10 2019."},{"key":"bibr304-11795735221092517","unstructured":"Sage Therapeutics to Host Sage Science Spotlight: SAGE-718 In Depth [press release]. May 13 2021."},{"key":"bibr305-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(14)70262-5"},{"key":"bibr306-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1515\/revneuro-2013-0040"},{"key":"bibr307-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1097\/00004850-200411000-00004"},{"key":"bibr308-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.20018"},{"key":"bibr309-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1002\/mds.23236"},{"key":"bibr310-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s00702-010-0430-7"},{"key":"bibr311-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1155\/2016\/8653132"},{"key":"bibr312-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuropharm.2008.07.037"},{"key":"bibr313-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/0091-3057(91)90386-G"},{"key":"bibr314-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-016-8145-9"},{"key":"bibr315-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2017.04.005"},{"key":"bibr316-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnagi.2019.00059"},{"key":"bibr317-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/nn.4338"},{"key":"bibr318-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1177\/0891988710383573"},{"key":"bibr319-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-013-0244-z"},{"key":"bibr320-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/j.1755-5949.2010.00134.x"},{"key":"bibr321-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2013.00114"},{"key":"bibr322-11795735221092517","doi-asserted-by":"publisher","DOI":"10.2217\/fnl.10.41"},{"key":"bibr323-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.pneurobio.2007.01.003"},{"key":"bibr324-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.1637-10.2010"},{"key":"bibr325-11795735221092517","doi-asserted-by":"publisher","DOI":"10.7150\/ijbs.4679"},{"key":"bibr326-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/emboj.2012.65"},{"key":"bibr327-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2015.157"},{"key":"bibr328-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0408936102"},{"key":"bibr329-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2009.200"},{"key":"bibr330-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.drudis.2014.02.006"},{"key":"bibr331-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3233\/JHD-130082"},{"key":"bibr332-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(15)00177-5"},{"key":"bibr333-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70178-4"},{"key":"bibr334-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2020.105199"},{"key":"bibr335-11795735221092517","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.603594"},{"key":"bibr336-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-020-75229-9"},{"key":"bibr337-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1093\/braincomms\/fcaa110"},{"key":"bibr338-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2018.09.001"},{"key":"bibr339-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1111\/ene.13413"},{"key":"bibr340-11795735221092517","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2009.180"},{"key":"bibr341-11795735221092517","volume-title":"Neurobiology of Huntington's Disease: Applications to Drug Discovery","author":"Meghan S","year":"2011"}],"container-title":["Journal of Central Nervous System Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/11795735221092517","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/11795735221092517","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/11795735221092517","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T15:59:33Z","timestamp":1777391973000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/11795735221092517"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,4]]},"references-count":341,"alternative-id":["10.1177\/11795735221092517"],"URL":"https:\/\/doi.org\/10.1177\/11795735221092517","relation":{},"ISSN":["1179-5735","1179-5735"],"issn-type":[{"value":"1179-5735","type":"print"},{"value":"1179-5735","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,4]]},"article-number":"11795735221092517"}}