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Mitochondrial malic enzyme reduced

MedGen UID:
383965
Concept ID:
C1856697
Finding
Synonym: Decreased mitochondrial malic enzyme
 
HPO: HP:0003232

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVMitochondrial malic enzyme reduced

Conditions with this feature

Friedreich ataxia 1
MedGen UID:
383962
Concept ID:
C1856689
Disease or Syndrome
Typical Friedreich ataxia (FRDA) is characterized by progressive ataxia with onset from early childhood to early adulthood with mean age at onset from 10 to 15 years (range: age two years to the eighth decade). Ataxia, manifesting initially as poor balance when walking, is typically followed by upper-limb ataxia, dysarthria, dysphagia, peripheral motor and sensory neuropathy, spasticity, autonomic disturbance, and often abnormal eye movements and optic atrophy. Hypertrophic cardiomyopathy is present in about two thirds of individuals; occasionally it is diagnosed prior to the onset of ataxia. Diabetes mellitus and impaired glucose tolerance can also occur. Among individuals with FRDA, about 75% have "typical Friedreich ataxia" and about 25% of individuals with biallelic FXN full-penetrance GAA repeat expansions have "atypical Friedreich ataxia" that includes late-onset FRDA (LOFA) (i.e., onset after age 25 years), very late-onset FRDA (VLOFA) (i.e., onset after age 40 years), and FRDA with retained reflexes (FARR).
Friedreich ataxia 2
MedGen UID:
356134
Concept ID:
C1865981
Disease or Syndrome
Typical Friedreich ataxia (FRDA) is characterized by progressive ataxia with onset from early childhood to early adulthood with mean age at onset from 10 to 15 years (range: age two years to the eighth decade). Ataxia, manifesting initially as poor balance when walking, is typically followed by upper-limb ataxia, dysarthria, dysphagia, peripheral motor and sensory neuropathy, spasticity, autonomic disturbance, and often abnormal eye movements and optic atrophy. Hypertrophic cardiomyopathy is present in about two thirds of individuals; occasionally it is diagnosed prior to the onset of ataxia. Diabetes mellitus and impaired glucose tolerance can also occur. Among individuals with FRDA, about 75% have "typical Friedreich ataxia" and about 25% of individuals with biallelic FXN full-penetrance GAA repeat expansions have "atypical Friedreich ataxia" that includes late-onset FRDA (LOFA) (i.e., onset after age 25 years), very late-onset FRDA (VLOFA) (i.e., onset after age 40 years), and FRDA with retained reflexes (FARR).

Professional guidelines

PubMed

Kang JH, Lee SH, Lee JS, Nam B, Seong TW, Son J, Jang H, Hong KM, Lee C, Kim SY
Oncotarget 2016 Aug 2;7(31):49397-49410. doi: 10.18632/oncotarget.10354. PMID: 27384481Free PMC Article

Recent clinical studies

Etiology

Schöpf B, Weissensteiner H, Schäfer G, Fazzini F, Charoentong P, Naschberger A, Rupp B, Fendt L, Bukur V, Giese I, Sorn P, Sant'Anna-Silva AC, Iglesias-Gonzalez J, Sahin U, Kronenberg F, Gnaiger E, Klocker H
Nat Commun 2020 Mar 20;11(1):1487. doi: 10.1038/s41467-020-15237-5. PMID: 32198407Free PMC Article
Kang JH, Lee SH, Lee JS, Nam B, Seong TW, Son J, Jang H, Hong KM, Lee C, Kim SY
Oncotarget 2016 Aug 2;7(31):49397-49410. doi: 10.18632/oncotarget.10354. PMID: 27384481Free PMC Article

Diagnosis

Del Prado L, Jaraíz-Rodríguez M, Agro M, Zamora-Dorta M, Azpiazu N, Calleja M, Lopez-Manzaneda M, de Juan-Sanz J, Fernández-Rodrigo A, Esteban JA, Girona M, Quintana A, Balsa E
Nat Commun 2024 Oct 7;15(1):8682. doi: 10.1038/s41467-024-52968-1. PMID: 39375345Free PMC Article
Koch J, Broeks MH, Gautschi M, Jans J, Laemmle A
Mol Genet Metab 2024 Aug;142(4):108520. Epub 2024 Jun 24 doi: 10.1016/j.ymgme.2024.108520. PMID: 38945121
Ren JG, Seth P, Clish CB, Lorkiewicz PK, Higashi RM, Lane AN, Fan TW, Sukhatme VP
Sci Rep 2014 Jun 24;4:5414. doi: 10.1038/srep05414. PMID: 24957098Free PMC Article
Yin F, Sancheti H, Cadenas E
Antioxid Redox Signal 2012 Dec 15;17(12):1714-27. Epub 2012 Jun 11 doi: 10.1089/ars.2012.4639. PMID: 22530585Free PMC Article
Ichiki T, Tanaka M, Nishikimi M, Suzuki H, Ozawa T, Kobayashi M, Wada Y
Ann Neurol 1988 Mar;23(3):287-94. doi: 10.1002/ana.410230312. PMID: 3132077

Therapy

Sarfraz I, Rasul A, Hussain G, Hussain SM, Ahmad M, Nageen B, Jabeen F, Selamoglu Z, Ali M
IUBMB Life 2018 Nov;70(11):1076-1083. Epub 2018 Aug 29 doi: 10.1002/iub.1930. PMID: 30160039
Kang JH, Lee SH, Lee JS, Nam B, Seong TW, Son J, Jang H, Hong KM, Lee C, Kim SY
Oncotarget 2016 Aug 2;7(31):49397-49410. doi: 10.18632/oncotarget.10354. PMID: 27384481Free PMC Article
Bescós R, Sureda A, Tur JA, Pons A
Sports Med 2012 Feb 1;42(2):99-117. doi: 10.2165/11596860-000000000-00000. PMID: 22260513

Prognosis

Sarfraz I, Rasul A, Hussain G, Hussain SM, Ahmad M, Nageen B, Jabeen F, Selamoglu Z, Ali M
IUBMB Life 2018 Nov;70(11):1076-1083. Epub 2018 Aug 29 doi: 10.1002/iub.1930. PMID: 30160039

Clinical prediction guides

Del Prado L, Jaraíz-Rodríguez M, Agro M, Zamora-Dorta M, Azpiazu N, Calleja M, Lopez-Manzaneda M, de Juan-Sanz J, Fernández-Rodrigo A, Esteban JA, Girona M, Quintana A, Balsa E
Nat Commun 2024 Oct 7;15(1):8682. doi: 10.1038/s41467-024-52968-1. PMID: 39375345Free PMC Article
Ryan EM, Sadiku P, Coelho P, Watts ER, Zhang A, Howden AJM, Sanchez-Garcia MA, Bewley M, Cole J, McHugh BJ, Vermaelen W, Ghesquiere B, Carmeliet P, Rodriguez Blanco G, Von Kriegsheim A, Sanchez Y, Rumsey W, Callahan JF, Cooper G, Parkinson N, Baillie K, Cantrell DA, McCafferty J, Choudhury G, Singh D, Dockrell DH, Whyte MKB, Walmsley SR
Am J Respir Crit Care Med 2023 Apr 15;207(8):998-1011. doi: 10.1164/rccm.202203-0482OC. PMID: 36724365Free PMC Article
Matheny RW Jr, Kolb AL, Geddis AV, Roberts BM
Physiol Rep 2022 Nov;10(21):e15481. doi: 10.14814/phy2.15481. PMID: 36325583Free PMC Article
Hsieh JY, Liu GY, Chang GG, Hung HC
J Biol Chem 2006 Aug 11;281(32):23237-45. Epub 2006 Jun 6 doi: 10.1074/jbc.M603451200. PMID: 16757477
Unnikrishnan LS, Raj RK
Int J Parasitol 1995 Jul;25(7):779-85. doi: 10.1016/0020-7519(94)00210-f. PMID: 7558563

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