Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
Infobox references
Chemical compound
Acetylleucine is a modified amino acid used in the treatment of vertigo[2] and cerebellar ataxia.
Acetylleucine is also being developed as a possible treatment for several neurological disorders by IntraBio Inc.[3] Clinical trials with acetylleucine for the treatment of three orphan, fatal, neurodegenerative disorders are underway: Niemann-Pick disease type C,[4] GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases),[5] and ataxia–telangiectasia.[6] In 2020, IntraBio announced the successful multinational clinical trial results of the Niemann-Pick type C clinical trial.[7] IntraBio is also investigating acetylleucine for the treatment of common inherited and acquired neurological diseases including Lewy body dementia,[8] amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine[9] Acetylleucine has received orphan drug designations from the U.S. Food & Drug Administration (FDA)[10][11][12][13] and the European Commission.[14][15][16][17]
^"N-Acetyl-DL-leucine". PubChem Open Chemistry Database. Retrieved 26 Mar 2017.
^"N07CA04 (acetylleucine)". WHO Collaborating Centre for Drug Statistics Methodology. Norwegian Institute of Public Health. 19 Dec 2016. Retrieved 26 Mar 2017.
^"IntraBio". Archived from the original on 2019-08-01. Retrieved 2019-08-01.
^"N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) - Full Text View - ClinicalTrials.gov". clinicaltrials.gov. Retrieved 2019-08-01.
^"N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease) - Full Text View - ClinicalTrials.gov". clinicaltrials.gov. Retrieved 2019-08-01.
^"N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) - Full Text View - ClinicalTrials.gov". clinicaltrials.gov. Retrieved 2019-08-01.
^"IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C". intrabio.com. 19 October 2020. Retrieved 2021-08-01.
^Passmore, Peter (2014-04-15). "A clinical trial to test amlodipine as a new treatment for vascular dementia". doi:10.1186/isrctn31208535. {{cite journal}}: Cite journal requires |journal= (help)
^Strupp, Michael; Bayer, Otmar; Feil, Katharina; Straube, Andreas (2019-02-01). "Prophylactic treatment of migraine with and without aura with acetyl-dl-leucine: a case series". Journal of Neurology. 266 (2): 525–529. doi:10.1007/s00415-018-9155-6. ISSN 1432-1459. PMID 30547273. S2CID 56148131.
^"Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2019-08-03.
^"Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2019-08-03.
^"Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2019-08-03.
^"Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2019-08-03.
^"Public Health - European Commission". Union Register of medicinal products. Retrieved 2019-08-03.
^"Public Health - European Commission". Union Register of medicinal products. Retrieved 2019-08-03.
^"Public Health - European Commission". Union Register of medicinal products. Retrieved 2019-08-03.
^"Public Health - European Commission". Union Register of medicinal products. Retrieved 2019-08-03.
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