U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Leigh disease

MedGen UID:
44095
Concept ID:
C0023264
Disease or Syndrome
Synonyms: Disease, Leigh's; Encephalomyelitides, Subacute Necrotizing; Encephalomyelitis, Subacute Necrotizing; Encephalomyelopathies, Subacute Necrotizing; Encephalomyelopathy, Subacute Necrotizing; Encephalopathies, Subacute Necrotizing; Encephalopathy, Subacute Necrotizing; Leigh Disease; Leigh Syndrome; Leigh's Disease; Leighs Disease; Necrotizing Encephalomyelitides, Subacute; Necrotizing Encephalomyelitis, Subacute; Necrotizing Encephalomyelopathies, Subacute; Necrotizing Encephalomyelopathy, Subacute; Necrotizing Encephalopathies, Subacute; Necrotizing Encephalopathy, Subacute; Subacute Necrotizing Encephalomyelitides; Subacute Necrotizing Encephalomyelitis; Subacute Necrotizing Encephalomyelopathies; Subacute Necrotizing Encephalomyelopathy; Subacute Necrotizing Encephalopathies; Subacute Necrotizing Encephalopathy
SNOMED CT: SNEM - Subacute necrotizing encephalomyelopathy (29570005); Leigh syndrome (29570005); Leigh disease (29570005); Leighs disease (29570005); Leigh's disease (29570005); Subacute necrotizing encephalopathy (29570005); Subacute necrotizing encephalomyelopathy (29570005)
 
OMIM®: 590060; 590095

Definition

An inherited disorder affecting the nervous system, caused by genetic mutations in the mitochondrial DNA or deficiency of pyruvate dehydrogenase. Signs and symptoms appear in infancy and include loss of the motor abilities, poor sucking abilities, irritability, lack of muscle tone, and seizures. [from NCI]

Term Hierarchy

Professional guidelines

PubMed

Thorburn DR, Dahl HH
Am J Med Genet 2001 Spring;106(1):102-14. doi: 10.1002/ajmg.1380. PMID: 11579429

Recent clinical studies

Etiology

Pizzamiglio C, Hanna MG, Pitceathly RDS
Handb Clin Neurol 2024;204:53-76. doi: 10.1016/B978-0-323-99209-1.00004-1. PMID: 39322395
McCormick EM, Keller K, Taylor JP, Coffey AJ, Shen L, Krotoski D, Harding B; NICHD ClinGen U24 Mitochondrial Disease Gene Curation Expert Panel, Gai X, Falk MJ, Zolkipli-Cunningham Z, Rahman S
Ann Neurol 2023 Oct;94(4):696-712. Epub 2023 Aug 12 doi: 10.1002/ana.26716. PMID: 37255483Free PMC Article
van de Wal MAE, Adjobo-Hermans MJW, Keijer J, Schirris TJJ, Homberg JR, Wieckowski MR, Grefte S, van Schothorst EM, van Karnebeek C, Quintana A, Koopman WJH
Brain 2022 Mar 29;145(1):45-63. doi: 10.1093/brain/awab426. PMID: 34849584Free PMC Article
Lim AZ, Ng YS, Blain A, Jiminez-Moreno C, Alston CL, Nesbitt V, Simmons L, Santra S, Wassmer E, Blakely EL, Turnbull DM, Taylor RW, Gorman GS, McFarland R
Ann Neurol 2022 Jan;91(1):117-130. Epub 2021 Nov 12 doi: 10.1002/ana.26260. PMID: 34716721Free PMC Article
Robinson BH
Pediatr Res 2000 Nov;48(5):581-5. doi: 10.1203/00006450-200011000-00004. PMID: 11044474

Diagnosis

Rahman S
Handb Clin Neurol 2023;194:43-63. doi: 10.1016/B978-0-12-821751-1.00015-4. PMID: 36813320
Stenton SL, Zou Y, Cheng H, Liu Z, Wang J, Shen D, Jin H, Ding C, Tang X, Sun S, Han H, Ma Y, Zhang W, Jin R, Wang H, Sun D, Lv JL, Prokisch H, Fang F
Ann Neurol 2022 Apr;91(4):466-482. Epub 2022 Mar 6 doi: 10.1002/ana.26313. PMID: 35094435
Finsterer J, Scorza FA, Fiorini AC, Scorza CA
Pediatr Neurol 2020 Sep;110:25-29. Epub 2020 Jul 16 doi: 10.1016/j.pediatrneurol.2020.03.009. PMID: 32684373
Lake NJ, Compton AG, Rahman S, Thorburn DR
Ann Neurol 2016 Feb;79(2):190-203. Epub 2015 Dec 15 doi: 10.1002/ana.24551. PMID: 26506407
Baertling F, Rodenburg RJ, Schaper J, Smeitink JA, Koopman WJ, Mayatepek E, Morava E, Distelmaier F
J Neurol Neurosurg Psychiatry 2014 Mar;85(3):257-65. Epub 2013 Jun 14 doi: 10.1136/jnnp-2012-304426. PMID: 23772060

Therapy

Puighermanal E, Luna-Sánchez M, Gella A, van der Walt G, Urpi A, Royo M, Tena-Morraja P, Appiah I, de Donato MH, Menardy F, Bianchi P, Esteve-Codina A, Rodríguez-Pascau L, Vergara C, Gómez-Pallarès M, Marsicano G, Bellocchio L, Martinell M, Sanz E, Jurado S, Soriano FX, Pizcueta P, Quintana A
Nat Commun 2024 Sep 4;15(1):7730. doi: 10.1038/s41467-024-51884-8. PMID: 39231983Free PMC Article
Zilber S, Woleben K, Johnson SC, de Souza CFM, Boyce D, Freiert K, Boggs C, Messahel S, Burnworth MJ, Afolabi TM, Kayani S
Orphanet J Rare Dis 2023 Sep 4;18(1):264. doi: 10.1186/s13023-023-02886-0. PMID: 37667390Free PMC Article
Murali CN, Soler-Alfonso C, Loomes KM, Shah AA, Monteil D, Padilla CD, Scaglia F, Ganetzky R
Mol Genet Metab 2021 Feb;132(2):146-153. Epub 2021 Jan 14 doi: 10.1016/j.ymgme.2021.01.005. PMID: 33485800Free PMC Article
Jain IH, Zazzeron L, Goli R, Alexa K, Schatzman-Bone S, Dhillon H, Goldberger O, Peng J, Shalem O, Sanjana NE, Zhang F, Goessling W, Zapol WM, Mootha VK
Science 2016 Apr 1;352(6281):54-61. Epub 2016 Feb 25 doi: 10.1126/science.aad9642. PMID: 26917594Free PMC Article
Davis RE, Icke GC
Adv Clin Chem 1983;23:93-140. doi: 10.1016/s0065-2423(08)60399-6. PMID: 6398618

Prognosis

Stenton SL, Zou Y, Cheng H, Liu Z, Wang J, Shen D, Jin H, Ding C, Tang X, Sun S, Han H, Ma Y, Zhang W, Jin R, Wang H, Sun D, Lv JL, Prokisch H, Fang F
Ann Neurol 2022 Apr;91(4):466-482. Epub 2022 Mar 6 doi: 10.1002/ana.26313. PMID: 35094435
van de Wal MAE, Adjobo-Hermans MJW, Keijer J, Schirris TJJ, Homberg JR, Wieckowski MR, Grefte S, van Schothorst EM, van Karnebeek C, Quintana A, Koopman WJH
Brain 2022 Mar 29;145(1):45-63. doi: 10.1093/brain/awab426. PMID: 34849584Free PMC Article
Aldera AP, Govender D
J Clin Pathol 2018 Feb;71(2):95-97. Epub 2017 Oct 25 doi: 10.1136/jclinpath-2017-204677. PMID: 29070651
Baertling F, Rodenburg RJ, Schaper J, Smeitink JA, Koopman WJ, Mayatepek E, Morava E, Distelmaier F
J Neurol Neurosurg Psychiatry 2014 Mar;85(3):257-65. Epub 2013 Jun 14 doi: 10.1136/jnnp-2012-304426. PMID: 23772060
Pagliarini DJ, Calvo SE, Chang B, Sheth SA, Vafai SB, Ong SE, Walford GA, Sugiana C, Boneh A, Chen WK, Hill DE, Vidal M, Evans JG, Thorburn DR, Carr SA, Mootha VK
Cell 2008 Jul 11;134(1):112-23. doi: 10.1016/j.cell.2008.06.016. PMID: 18614015Free PMC Article

Clinical prediction guides

Stenton SL, Zou Y, Cheng H, Liu Z, Wang J, Shen D, Jin H, Ding C, Tang X, Sun S, Han H, Ma Y, Zhang W, Jin R, Wang H, Sun D, Lv JL, Prokisch H, Fang F
Ann Neurol 2022 Apr;91(4):466-482. Epub 2022 Mar 6 doi: 10.1002/ana.26313. PMID: 35094435
Lim AZ, Ng YS, Blain A, Jiminez-Moreno C, Alston CL, Nesbitt V, Simmons L, Santra S, Wassmer E, Blakely EL, Turnbull DM, Taylor RW, Gorman GS, McFarland R
Ann Neurol 2022 Jan;91(1):117-130. Epub 2021 Nov 12 doi: 10.1002/ana.26260. PMID: 34716721Free PMC Article
Baertling F, Rodenburg RJ, Schaper J, Smeitink JA, Koopman WJ, Mayatepek E, Morava E, Distelmaier F
J Neurol Neurosurg Psychiatry 2014 Mar;85(3):257-65. Epub 2013 Jun 14 doi: 10.1136/jnnp-2012-304426. PMID: 23772060
Pagliarini DJ, Calvo SE, Chang B, Sheth SA, Vafai SB, Ong SE, Walford GA, Sugiana C, Boneh A, Chen WK, Hill DE, Vidal M, Evans JG, Thorburn DR, Carr SA, Mootha VK
Cell 2008 Jul 11;134(1):112-23. doi: 10.1016/j.cell.2008.06.016. PMID: 18614015Free PMC Article
Carrozzo R, Piemonte F, Tessa A, Lucioli S, Rizza T, Meschini MC, Fattori F, Santorelli FM
Biosci Rep 2007 Jun;27(1-3):105-12. doi: 10.1007/s10540-007-9039-y. PMID: 17486440

Recent systematic reviews

Tiet MY, Lin Z, Gao F, Jennings MJ, Horvath R
J Neuromuscul Dis 2021;8(6):885-897. doi: 10.3233/JND-210715. PMID: 34308912Free PMC Article
Chang X, Wu Y, Zhou J, Meng H, Zhang W, Guo J
Medicine (Baltimore) 2020 Jan;99(5):e18634. doi: 10.1097/MD.0000000000018634. PMID: 32000367Free PMC Article

Supplemental Content

Table of contents

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.
    • Bookshelf
      See practice and clinical guidelines in NCBI Bookshelf. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...