{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,4]],"date-time":"2026-11-04T22:29:57Z","timestamp":1793831397634,"version":"4.6.1"},"reference-count":29,"publisher":"American Medical Association (AMA)","issue":"12","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Netw Open"],"DOI":"10.1001\/jamanetworkopen.2021.35841","type":"journal-article","created":{"date-parts":[[2021,12,14]],"date-time":"2021-12-14T16:49:51Z","timestamp":1639500591000},"page":"e2135841","source":"Crossref","is-referenced-by-count":44,"title":["Safety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia"],"prefix":"10.1001","volume":"4","author":[{"given":"Katharina","family":"Feil","sequence":"first","affiliation":[{"name":"Department of Neurology with Friedrich-Baur-Institute, Ludwig Maximilians University, University Hospital, Munich, Germany"},{"name":"German Center for Vertigo and Balance Disorders (DSGZ), Ludwig Maximilians University, University Hospital, Campus Grosshadern, Munich, Germany"},{"name":"Department of Neurology and Stroke, University Hospital T\u00fcbingen, T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Adrion","sequence":"additional","affiliation":[{"name":"Institute for Medical Informatics, Biometry and Epidemiology (IBE), Ludwig Maximilians University, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Boesch","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical University Innsbruck, Innsbruck, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Doss","sequence":"additional","affiliation":[{"name":"Department of Neurology, Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin, corporate member of Freie Universit\u00e4t Berlin, Humboldt-Universit\u00e4t zu Berlin, Berlin, Germany"},{"name":"Department of Neurological Sciences, University of Nebraska Medical Center (UNMC), Omaha"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Giordano","sequence":"additional","affiliation":[{"name":"German Center for Neurodegenerative Diseases (DZNE), Center for Clinical Research, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holger","family":"Hengel","sequence":"additional","affiliation":[{"name":"Department of Neurology and Hertie-Institute for Clinical Brain Research, University Hospital T\u00fcbingen, T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heike","family":"Jacobi","sequence":"additional","affiliation":[{"name":"German Center for Neurodegenerative Diseases (DZNE), Center for Clinical Research, Bonn, Germany"},{"name":"Department of Neurology, University Hospital Heidelberg, Heidelberg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Klockgether","sequence":"additional","affiliation":[{"name":"German Center for Neurodegenerative Diseases (DZNE), Center for Clinical Research, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Klopstock","sequence":"additional","affiliation":[{"name":"Department of Neurology with Friedrich-Baur-Institute, Ludwig Maximilians University, University Hospital, 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"}]},{"given":"Wolfgang","family":"Nachbauer","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical University Innsbruck, Innsbruck, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ludger","family":"Sch\u00f6ls","sequence":"additional","affiliation":[{"name":"Department of Neurology and Hertie-Institute for Clinical Brain Research, University Hospital T\u00fcbingen, T\u00fcbingen, Germany"},{"name":"German Center for Neurodegenerative Diseases (DZNE), T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katharina Marie","family":"Steiner","sequence":"additional","affiliation":[{"name":"Department of Neurology and Center for Translational Neuro- and Behavioral Sciences, University Hospital Essen, Essen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Stendel","sequence":"additional","affiliation":[{"name":"Department of Neurology with Friedrich-Baur-Institute, Ludwig Maximilians University, University Hospital, Munich, Germany"},{"name":"Munich Cluster for Systems Neurology (SyNergy), Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dagmar","family":"Timmann","sequence":"additional","affiliation":[{"name":"Department of Neurology and Center for Translational Neuro- and Behavioral Sciences, University Hospital Essen, Essen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivonne","family":"Naumann","sequence":"additional","affiliation":[{"name":"German Center for Vertigo and Balance Disorders (DSGZ), Ludwig Maximilians University, University Hospital, Campus Grosshadern, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrich","family":"Mansmann","sequence":"additional","affiliation":[{"name":"Institute for Medical Informatics, Biometry and Epidemiology (IBE), Ludwig Maximilians University, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Strupp","sequence":"additional","affiliation":[{"name":"Department of Neurology with Friedrich-Baur-Institute, Ludwig Maximilians University, University Hospital, Munich, Germany"},{"name":"German Center for Vertigo and Balance Disorders (DSGZ), Ludwig Maximilians University, University Hospital, Campus Grosshadern, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"ALCAT Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","published-online":{"date-parts":[[2021,12,14]]},"reference":[{"issue":"3","key":"zoi211007r1","doi-asserted-by":"publisher","first-page":"174","DOI":"10.1159\/000358801","article-title":"The global epidemiology of hereditary ataxia and spastic paraplegia: a systematic review of prevalence studies.","volume":"42","author":"Ruano","year":"2014","journal-title":"Neuroepidemiology"},{"issue":"3","key":"zoi211007r2","doi-asserted-by":"publisher","first-page":"369","DOI":"10.1007\/s12311-015-0687-3","article-title":"Consensus paper: revisiting the symptoms and signs of cerebellar syndrome.","volume":"15","author":"Bodranghien","year":"2016","journal-title":"Cerebellum"},{"issue":"1","key":"zoi211007r3","doi-asserted-by":"publisher","first-page":"24","DOI":"10.1038\/s41572-019-0074-3","article-title":"Spinocerebellar ataxia.","volume":"5","author":"Klockgether","year":"2019","journal-title":"Nat Rev Dis Primers"},{"issue":"4","key":"zoi211007r4","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1016\/S1474-4422(18)30042-5","article-title":"Survival in patients with spinocerebellar ataxia types 1, 2, 3, and 6 (EUROSCA): a longitudinal cohort study.","volume":"17","author":"Diallo","year":"2018","journal-title":"Lancet Neurol"},{"issue":"10","key":"zoi211007r5","doi-asserted-by":"publisher","first-page":"839","DOI":"10.1212\/WNL.0b013e3181d31e23","article-title":"Riluzole in cerebellar ataxia: a randomized, double-blind, placebo-controlled pilot trial.","volume":"74","author":"Ristori","year":"2010","journal-title":"Neurology"},{"issue":"10","key":"zoi211007r6","doi-asserted-by":"publisher","first-page":"985","DOI":"10.1016\/S1474-4422(15)00201-X","article-title":"Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial.","volume":"14","author":"Romano","year":"2015","journal-title":"Lancet Neurol"},{"issue":"8","key":"zoi211007r7","doi-asserted-by":"publisher","first-page":"545","DOI":"10.1212\/WNL.0b013e318247cc7a","article-title":"A randomized trial of varenicline (Chantix) for the treatment of spinocerebellar ataxia type 3.","volume":"78","author":"Zesiewicz","year":"2012","journal-title":"Neurology"},{"issue":"3","key":"zoi211007r8","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1136\/jnnp-2019-322168","article-title":"Effect of rovatirelin in patients with cerebellar ataxia: two randomised double-blind placebo-controlled phase 3 trials.","volume":"91","author":"Nishizawa","year":"2020","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"22","key":"zoi211007r9","doi-asserted-by":"publisher","first-page":"1823","DOI":"10.1212\/WNL.0b013e3181c33adf","article-title":"Intensive coordinative training improves motor performance in degenerative cerebellar disease.","volume":"73","author":"Ilg","year":"2009","journal-title":"Neurology"},{"issue":"4","key":"zoi211007r10","doi-asserted-by":"publisher","first-page":"1211","DOI":"10.1007\/s00415-020-09717-3","article-title":"The neurological update: therapies for cerebellar ataxias in 2020.","volume":"267","author":"Gandini","year":"2020","journal-title":"J Neurol"},{"issue":"1","key":"zoi211007r11","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1159\/000148206","article-title":"Effects of acetyl-DL-leucine in vestibular patients: a clinical study following neurotomy and labyrinthectomy.","volume":"14","author":"Ferber-Viart","year":"2009","journal-title":"Audiol Neurootol"},{"issue":"4","key":"zoi211007r12","doi-asserted-by":"publisher","first-page":"735","DOI":"10.1046\/j.0953-816x.2000.01447.x","article-title":"In vitro effects of acetyl-DL-leucine (tanganil) on central vestibular neurons and vestibulo-ocular networks of the guinea-pig.","volume":"13","author":"Vibert","year":"2001","journal-title":"Eur J Neurosci"},{"issue":"3","key":"zoi211007r13","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0120891","article-title":"N-acetyl-L-leucine accelerates vestibular compensation after unilateral labyrinthectomy by action in the cerebellum and thalamus.","volume":"10","author":"G\u00fcnther","year":"2015","journal-title":"PLoS One"},{"issue":"1","key":"zoi211007r14","doi-asserted-by":"publisher","first-page":"9249","DOI":"10.1038\/s41598-021-88693-8","article-title":"N-Acetyl-L-leucine improves functional recovery and attenuates cortical cell death and neuroinflammation after traumatic brain injury in mice.","volume":"11","author":"Hegdekar","year":"2021","journal-title":"Sci Rep"},{"key":"zoi211007r15","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1016\/S0079-6123(05)51006-9","article-title":"The anatomy of the vestibular nuclei.","volume":"151","author":"Highstein","year":"2006","journal-title":"Prog Brain Res"},{"issue":"4","key":"zoi211007r16","doi-asserted-by":"publisher","first-page":"1963","DOI":"10.1152\/jn.00334.2006","article-title":"Morphology and physiology of the cerebellar vestibulolateral lobe pathways linked to oculomotor function in the goldfish.","volume":"96","author":"Straka","year":"2006","journal-title":"J Neurophysiol"},{"issue":"10","key":"zoi211007r17","doi-asserted-by":"publisher","first-page":"2556","DOI":"10.1007\/s00415-013-7016-x","article-title":"Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series.","volume":"260","author":"Strupp","year":"2013","journal-title":"J Neurol"},{"key":"zoi211007r18","doi-asserted-by":"publisher","first-page":"8","DOI":"10.1186\/s40673-016-0046-2","article-title":"Acetyl-DL-leucine improves gait variability in patients with cerebellar ataxia-a case series.","volume":"3","author":"Schniepp","year":"2016","journal-title":"Cerebellum Ataxias"},{"issue":"16","key":"zoi211007r19","doi-asserted-by":"publisher","first-page":"1368","DOI":"10.1212\/WNL.0000000000002041","article-title":"Acetyl-DL-leucine in Niemann-Pick type C: a case series.","volume":"85","author":"Bremova","year":"2015","journal-title":"Neurology"},{"issue":"5","key":"zoi211007r20","doi-asserted-by":"publisher","first-page":"603","DOI":"10.1007\/s00228-018-02617-6","article-title":"Efficacy of acetylleucine in vertigo and dizziness: a systematic review of randomised controlled trials.","volume":"75","author":"Vanderkam","year":"2019","journal-title":"Eur J Clin Pharmacol"},{"issue":"1","key":"zoi211007r21","doi-asserted-by":"publisher","first-page":"7","DOI":"10.1186\/s12883-016-0786-x","article-title":"Effects of acetyl-DL-leucine on cerebellar ataxia (ALCAT trial): study protocol for a multicenter, multinational, randomized, double-blind, placebo-controlled, crossover phase III trial.","volume":"17","author":"Feil","year":"2017","journal-title":"BMC Neurol"},{"issue":"10","key":"zoi211007r22","doi-asserted-by":"publisher","first-page":"1727","DOI":"10.1007\/s11136-011-9903-x","article-title":"Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L).","volume":"20","author":"Herdman","year":"2011","journal-title":"Qual Life Res"},{"issue":"11","key":"zoi211007r23","doi-asserted-by":"publisher","first-page":"1717","DOI":"10.1212\/01.wnl.0000219042.60538.92","article-title":"Scale for the assessment and rating of ataxia: development of a new clinical scale.","volume":"66","author":"Schmitz-H\u00fcbsch","year":"2006","journal-title":"Neurology"},{"issue":"5","key":"zoi211007r24","doi-asserted-by":"publisher","first-page":"870","DOI":"10.1002\/mds.v26.5","article-title":"Depression comorbidity in spinocerebellar ataxia.","volume":"26","author":"Schmitz-H\u00fcbsch","year":"2011","journal-title":"Mov Disord"},{"issue":"11","key":"zoi211007r25","doi-asserted-by":"publisher","first-page":"953","DOI":"10.1212\/WNL.0b013e31821043a4","article-title":"Fatigue in spinocerebellar ataxia: patient self-assessment of an early and disabling symptom.","volume":"76","author":"Brusse","year":"2011","journal-title":"Neurology"},{"issue":"2","key":"zoi211007r26","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0229585","article-title":"Unexpected differences in the pharmacokinetics of N-acetyl-DL-leucine enantiomers after oral dosing and their clinical relevance.","volume":"15","author":"Churchill","year":"2020","journal-title":"PLoS One"},{"issue":"4","key":"zoi211007r27","doi-asserted-by":"crossref","DOI":"10.3390\/jcm9041050","article-title":"Beneficial effects of acetyl-DL-leucine (ADLL) in a mouse model of Sandhoff disease.","volume":"9","author":"Kaya","year":"2020","journal-title":"J Clin Med"},{"key":"zoi211007r28","doi-asserted-by":"publisher","first-page":"112","DOI":"10.1016\/j.jns.2019.04.039","article-title":"Nocebo in cerebellar ataxia: a systematic review and meta-analysis of placebo-controlled clinical trials.","volume":"401","author":"Alam","year":"2019","journal-title":"J Neurol Sci"},{"issue":"2","key":"zoi211007r29","doi-asserted-by":"publisher","first-page":"488","DOI":"10.1007\/s12311-011-0316-8","article-title":"Ataxia rating scales--psychometric profiles, natural history and their application in clinical trials.","volume":"11","author":"Saute","year":"2012","journal-title":"Cerebellum"}],"container-title":["JAMA Network Open"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jamanetworkopen\/articlepdf\/2787099\/feil_2021_oi_211007_1638886097.4648.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,14]],"date-time":"2021-12-14T16:49:55Z","timestamp":1639500595000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jamanetworkopen\/fullarticle\/2787099"}},"subtitle":["The ALCAT Randomized Clinical Crossover Trial"],"short-title":[],"issued":{"date-parts":[[2021,12,14]]},"references-count":29,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2021,12,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jamanetworkopen.2021.35841","relation":{},"ISSN":["2574-3805"],"issn-type":[{"value":"2574-3805","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,12,14]]}}}