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Coenzyme Q10 deficiency

MedGen UID:
334528
Concept ID:
C1843920
Disease or Syndrome
Synonyms: Coenzyme Q10 deficiency, primary; CoQ10 deficiency; CoQ10 deficiency, primary
SNOMED CT: Coenzyme Q10 deficiency (724575009)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Monarch Initiative: MONDO:0018151
OMIM® Phenotypic series: PS607426
Orphanet: ORPHA35656

Authors:
Leonardo Salviati  |  Eva Trevisson  |  Caterina Agosto, et. al.   view full author information

Additional description

From GeneReviews Overview
Primary coenzyme Q10 (CoQ10) deficiency is usually associated with multisystem involvement, including neurologic manifestations such as fatal neonatal encephalopathy with hypotonia; a late-onset slowly progressive multiple-system atrophy-like phenotype (neurodegeneration with autonomic failure and various combinations of parkinsonism and cerebellar ataxia, and pyramidal dysfunction); and dystonia, spasticity, seizures, and intellectual disability. Steroid-resistant nephrotic syndrome (SRNS), the hallmark renal manifestation, is often the initial manifestation either as isolated renal involvement that progresses to end-stage renal disease (ESRD), or associated with encephalopathy (seizures, stroke-like episodes, severe neurologic impairment) resulting in early death. Hypertrophic cardiomyopathy (HCM), retinopathy or optic atrophy, and sensorineural hearing loss can also be seen.

Professional guidelines

PubMed

Drovandi S, Lipska-Ziętkiewicz BS, Ozaltin F, Emma F, Gulhan B, Boyer O, Trautmann A, Ziętkiewicz S, Xu H, Shen Q, Rao J, Riedhammer KM, Heemann U, Hoefele J, Stenton SL, Tsygin AN, Ng KH, Fomina S, Benetti E, Aurelle M, Prikhodina L, Schijvens AM, Tabatabaeifar M, Jankowski M, Baiko S, Mao J, Feng C, Deng F, Rousset-Rouviere C, Stańczyk M, Bałasz-Chmielewska I, Fila M, Durkan AM, Levart TK, Dursun I, Esfandiar N, Haas D, Bjerre A, Anarat A, Benz MR, Talebi S, Hooman N, Ariceta G; PodoNet Consortium; mitoNET Consortium; CCGKDD Consortium, Schaefer F
Kidney Int 2022 Sep;102(3):592-603. Epub 2022 Apr 26 doi: 10.1016/j.kint.2022.02.040. PMID: 35483523
Desbats MA, Morbidoni V, Silic-Benussi M, Doimo M, Ciminale V, Cassina M, Sacconi S, Hirano M, Basso G, Pierrel F, Navas P, Salviati L, Trevisson E
Hum Mol Genet 2016 Oct 1;25(19):4256-4265. Epub 2016 Aug 4 doi: 10.1093/hmg/ddw257. PMID: 27493029
Hassani A, Horvath R, Chinnery PF
Curr Opin Neurol 2010 Oct;23(5):459-65. doi: 10.1097/WCO.0b013e32833d1096. PMID: 20651591

Recent clinical studies

Etiology

Al Saadi T, Assaf Y, Farwati M, Turkmani K, Al-Mouakeh A, Shebli B, Khoja M, Essali A, Madmani ME
Cochrane Database Syst Rev 2021 Feb 3;(2)(2):CD008684. doi: 10.1002/14651858.CD008684.pub3. PMID: 35608922Free PMC Article
Martinefski MR, Yamasato MF, Di Carlo MB, Daruich JR, Tripodi VP
Clin Res Hepatol Gastroenterol 2021 Nov;45(6):101624. Epub 2021 Mar 4 doi: 10.1016/j.clinre.2021.101624. PMID: 33676282
Stamelou M, Bhatia KP
Curr Opin Neurol 2016 Aug;29(4):480-5. doi: 10.1097/WCO.0000000000000355. PMID: 27272977
Desbats MA, Lunardi G, Doimo M, Trevisson E, Salviati L
J Inherit Metab Dis 2015 Jan;38(1):145-56. Epub 2014 Aug 5 doi: 10.1007/s10545-014-9749-9. PMID: 25091424
Jayadev S, Bird TD
Genet Med 2013 Sep;15(9):673-83. Epub 2013 Mar 28 doi: 10.1038/gim.2013.28. PMID: 23538602

Diagnosis

Diessl J, Berndtsson J, Broeskamp F, Habernig L, Kohler V, Vazquez-Calvo C, Nandy A, Peselj C, Drobysheva S, Pelosi L, Vögtle FN, Pierrel F, Ott M, Büttner S
Nat Commun 2022 Oct 13;13(1):6061. doi: 10.1038/s41467-022-33641-x. PMID: 36229432Free PMC Article
Boltshauser E, Weber KP
Handb Clin Neurol 2018;154:287-298. doi: 10.1016/B978-0-444-63956-1.00017-5. PMID: 29903445
Yubero D, Montero R, Santos-Ocaña C, Salviati L, Navas P, Artuch R
Expert Rev Mol Diagn 2018 Jun;18(6):491-498. Epub 2018 May 30 doi: 10.1080/14737159.2018.1478290. PMID: 29781757
Yubero D, Montero R, Armstrong J, Espinós C, Palau F, Santos-Ocaña C, Salviati L, Navas P, Artuch R
Expert Rev Mol Diagn 2015;15(8):1049-59. Epub 2015 Jul 4 doi: 10.1586/14737159.2015.1062727. PMID: 26144946
Jayadev S, Bird TD
Genet Med 2013 Sep;15(9):673-83. Epub 2013 Mar 28 doi: 10.1038/gim.2013.28. PMID: 23538602

Therapy

Al Saadi T, Assaf Y, Farwati M, Turkmani K, Al-Mouakeh A, Shebli B, Khoja M, Essali A, Madmani ME
Cochrane Database Syst Rev 2021 Feb 3;(2)(2):CD008684. doi: 10.1002/14651858.CD008684.pub3. PMID: 35608922Free PMC Article
Aaseth J, Alexander J, Alehagen U
Mech Ageing Dev 2021 Jul;197:111521. Epub 2021 Jun 12 doi: 10.1016/j.mad.2021.111521. PMID: 34129891
Awad AM, Bradley MC, Fernández-Del-Río L, Nag A, Tsui HS, Clarke CF
Essays Biochem 2018 Jul 20;62(3):361-376. doi: 10.1042/EBC20170106. PMID: 29980630Free PMC Article
Stamelou M, Bhatia KP
Curr Opin Neurol 2016 Aug;29(4):480-5. doi: 10.1097/WCO.0000000000000355. PMID: 27272977
Quinzii CM, DiMauro S, Hirano M
Neurochem Res 2007 Apr-May;32(4-5):723-7. Epub 2006 Nov 10 doi: 10.1007/s11064-006-9190-z. PMID: 17094036Free PMC Article

Prognosis

Cordts I, Semmler L, Prasuhn J, Seibt A, Herebian D, Navaratnarajah T, Park J, Deininger N, Laugwitz L, Göricke SL, Lingor P, Brüggemann N, Münchau A, Synofzik M, Timmann D, Mayr JA, Haack TB, Distelmaier F, Deschauer M
Mov Disord 2022 Oct;37(10):2147-2153. Epub 2022 Sep 1 doi: 10.1002/mds.29167. PMID: 36047608
Prasuhn J, Göttlich M, Ebeling B, Bodemann C, Großer S, Wellach I, Reuther K, Hanssen H, Brüggemann N
Parkinsonism Relat Disord 2022 Jun;99:91-95. Epub 2022 May 19 doi: 10.1016/j.parkreldis.2022.05.008. PMID: 35642996
Alehagen U, Aaseth J
J Trace Elem Med Biol 2015;31:157-62. Epub 2014 Nov 27 doi: 10.1016/j.jtemb.2014.11.006. PMID: 25511910
Desguerre I, Hully M, Rio M, Nabbout R
Rev Neurol (Paris) 2014 May;170(5):375-80. Epub 2014 May 5 doi: 10.1016/j.neurol.2014.03.010. PMID: 24810279
Palau F, Espinós C
Orphanet J Rare Dis 2006 Nov 17;1:47. doi: 10.1186/1750-1172-1-47. PMID: 17112370Free PMC Article

Clinical prediction guides

Gasmi A, Bjørklund G, Mujawdiya PK, Semenova Y, Piscopo S, Peana M
Crit Rev Food Sci Nutr 2024;64(12):3907-3919. Epub 2022 Oct 27 doi: 10.1080/10408398.2022.2137724. PMID: 36300654
Drovandi S, Lipska-Ziętkiewicz BS, Ozaltin F, Emma F, Gulhan B, Boyer O, Trautmann A, Ziętkiewicz S, Xu H, Shen Q, Rao J, Riedhammer KM, Heemann U, Hoefele J, Stenton SL, Tsygin AN, Ng KH, Fomina S, Benetti E, Aurelle M, Prikhodina L, Schijvens AM, Tabatabaeifar M, Jankowski M, Baiko S, Mao J, Feng C, Deng F, Rousset-Rouviere C, Stańczyk M, Bałasz-Chmielewska I, Fila M, Durkan AM, Levart TK, Dursun I, Esfandiar N, Haas D, Bjerre A, Anarat A, Benz MR, Talebi S, Hooman N, Ariceta G; PodoNet Consortium; mitoNET Consortium; CCGKDD Consortium, Schaefer F
Kidney Int 2022 Sep;102(3):592-603. Epub 2022 Apr 26 doi: 10.1016/j.kint.2022.02.040. PMID: 35483523
Martinefski MR, Yamasato MF, Di Carlo MB, Daruich JR, Tripodi VP
Clin Res Hepatol Gastroenterol 2021 Nov;45(6):101624. Epub 2021 Mar 4 doi: 10.1016/j.clinre.2021.101624. PMID: 33676282
Lucangioli S, Martinefski M, Tripodi V
Front Biosci (Schol Ed) 2016 Jun 1;8(2):321-30. doi: 10.2741/s466. PMID: 27100710
Desbats MA, Lunardi G, Doimo M, Trevisson E, Salviati L
J Inherit Metab Dis 2015 Jan;38(1):145-56. Epub 2014 Aug 5 doi: 10.1007/s10545-014-9749-9. PMID: 25091424

Recent systematic reviews

Wang Y, Hekimi S
J Cell Mol Med 2022 Sep;26(17):4635-4644. Epub 2022 Aug 19 doi: 10.1111/jcmm.17488. PMID: 35985679Free PMC Article
Drovandi S, Lipska-Ziętkiewicz BS, Ozaltin F, Emma F, Gulhan B, Boyer O, Trautmann A, Ziętkiewicz S, Xu H, Shen Q, Rao J, Riedhammer KM, Heemann U, Hoefele J, Stenton SL, Tsygin AN, Ng KH, Fomina S, Benetti E, Aurelle M, Prikhodina L, Schijvens AM, Tabatabaeifar M, Jankowski M, Baiko S, Mao J, Feng C, Deng F, Rousset-Rouviere C, Stańczyk M, Bałasz-Chmielewska I, Fila M, Durkan AM, Levart TK, Dursun I, Esfandiar N, Haas D, Bjerre A, Anarat A, Benz MR, Talebi S, Hooman N, Ariceta G; PodoNet Consortium; mitoNET Consortium; CCGKDD Consortium, Schaefer F
Kidney Int 2022 Sep;102(3):592-603. Epub 2022 Apr 26 doi: 10.1016/j.kint.2022.02.040. PMID: 35483523
Al Saadi T, Assaf Y, Farwati M, Turkmani K, Al-Mouakeh A, Shebli B, Khoja M, Essali A, Madmani ME
Cochrane Database Syst Rev 2021 Feb 3;(2)(2):CD008684. doi: 10.1002/14651858.CD008684.pub3. PMID: 35608922Free PMC Article
Madmani ME, Yusuf Solaiman A, Tamr Agha K, Madmani Y, Shahrour Y, Essali A, Kadro W
Cochrane Database Syst Rev 2014 Jun 2;(6):CD008684. doi: 10.1002/14651858.CD008684.pub2. PMID: 24049047
Ho MJ, Bellusci A, Wright JM
Cochrane Database Syst Rev 2009 Oct 7;(4):CD007435. doi: 10.1002/14651858.CD007435.pub2. PMID: 19821418

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