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Maternally-inherited mitochondrial myopathy

MedGen UID:
1800436
Concept ID:
C5569013
Disease or Syndrome
Synonyms: Maternally inherited mitochondrial myopathy; Mitochondrial DNA-related mitochondrial myopathy; mtDNA-related mitochondrial myopathy
SNOMED CT: Maternally inherited mitochondrial myopathy (1187517009)
Modes of inheritance:
Mitochondrial inheritance
MedGen UID:
165802
Concept ID:
C0887941
Genetic Function
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on the mitochondrial genome. Because the mitochondrial genome is essentially always maternally inherited, a mitochondrial condition can only be transmitted by females, although the condition can affect both sexes. The proportion of mutant mitochondria can vary (heteroplasmy).
 
Orphanet: ORPHA254788

Definition

A group of rare mitochondrial oxidative phosphorylation disorders due to mitochondrial DNA anomalies characterized by progressive, most commonly proximal, myopathy with variable degrees of weakness, exercise-induced muscle pain, and fatigue. Progressive external ophthalmoplegia is often observed. Additional features include neurological signs and symptoms (such as seizures, stroke-like episodes, or developmental delay), cardiomyopathy, involvement of liver, kidneys, and gastrointestinal tract, and diabetes. Lactic acidosis is frequently present, while recurrent rhabdomyolysis and myoglobinuria are rare. Muscle biopsy may reveal the presence of ragged-red fibers and a mosaic pattern of cytochrome c oxidase-negative fibers. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • Maternally-inherited mitochondrial myopathy

Professional guidelines

PubMed

Robinson KN, Terrazas S, Giordano-Mooga S, Xavier NA
Endocr Pract 2020 Feb;26(2):241-246. Epub 2019 Nov 4 doi: 10.4158/EP-2019-0270. PMID: 31682520
El-Hattab AW, Adesina AM, Jones J, Scaglia F
Mol Genet Metab 2015 Sep-Oct;116(1-2):4-12. Epub 2015 Jun 15 doi: 10.1016/j.ymgme.2015.06.004. PMID: 26095523
Thambisetty M, Newman NJ
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Recent clinical studies

Etiology

Ferreira F, Gonçalves Bacelar C, Lisboa-Gonçalves P, Paulo N, Quental R, Nunes AT, Silva R, Tavares I
Nefrologia (Engl Ed) 2023 Dec;43 Suppl 2:1-7. doi: 10.1016/j.nefroe.2024.01.017. PMID: 38355238
Yang M, Xu L, Xu C, Cui Y, Jiang S, Dong J, Liao L
Front Endocrinol (Lausanne) 2021;12:728043. Epub 2021 Nov 25 doi: 10.3389/fendo.2021.728043. PMID: 34899594Free PMC Article
Behjati M, Sabri MR, Etemadi Far M, Nejati M
Heart Fail Rev 2021 Mar;26(2):391-403. doi: 10.1007/s10741-020-10009-1. PMID: 32728985
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DiMauro S, Moraes CT
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Diagnosis

Ferreira F, Gonçalves Bacelar C, Lisboa-Gonçalves P, Paulo N, Quental R, Nunes AT, Silva R, Tavares I
Nefrologia (Engl Ed) 2023 Dec;43 Suppl 2:1-7. doi: 10.1016/j.nefroe.2024.01.017. PMID: 38355238
Behjati M, Sabri MR, Etemadi Far M, Nejati M
Heart Fail Rev 2021 Mar;26(2):391-403. doi: 10.1007/s10741-020-10009-1. PMID: 32728985
El-Hattab AW, Scaglia F
Cell Calcium 2016 Sep;60(3):199-206. Epub 2016 Mar 4 doi: 10.1016/j.ceca.2016.03.003. PMID: 26996063
Zeviani M, Tiranti V, Piantadosi C
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DiMauro S, Moraes CT
Arch Neurol 1993 Nov;50(11):1197-208. doi: 10.1001/archneur.1993.00540110075008. PMID: 8215979

Therapy

Finsterer J
Expert Opin Pharmacother 2019 Jul;20(10):1289-1297. Epub 2019 May 7 doi: 10.1080/14656566.2019.1609941. PMID: 31063406
Johnson SC, Martinez F, Bitto A, Gonzalez B, Tazaerslan C, Cohen C, Delaval L, Timsit J, Knebelmann B, Terzi F, Mahal T, Zhu Y, Morgan PG, Sedensky MM, Kaeberlein M, Legendre C, Suh Y, Canaud G
Kidney Int 2019 Feb;95(2):455-466. Epub 2018 Nov 22 doi: 10.1016/j.kint.2018.08.038. PMID: 30471880Free PMC Article
El-Hattab AW, Adesina AM, Jones J, Scaglia F
Mol Genet Metab 2015 Sep-Oct;116(1-2):4-12. Epub 2015 Jun 15 doi: 10.1016/j.ymgme.2015.06.004. PMID: 26095523
Moggio M, Colombo I, Peverelli L, Villa L, Xhani R, Testolin S, Di Mauro S, Sciacco M
Acta Myol 2014 Oct;33(2):86-93. PMID: 25709378Free PMC Article
El-Hattab AW, Emrick LT, Chanprasert S, Craigen WJ, Scaglia F
Int J Biochem Cell Biol 2014 Mar;48:85-91. Epub 2014 Jan 8 doi: 10.1016/j.biocel.2013.12.009. PMID: 24412347

Prognosis

Coussa RG, Parikh S, Traboulsi EI
Surv Ophthalmol 2021 Sep-Oct;66(5):838-855. Epub 2021 Feb 18 doi: 10.1016/j.survophthal.2021.02.008. PMID: 33610586
El-Hattab AW, Scaglia F
Cell Calcium 2016 Sep;60(3):199-206. Epub 2016 Mar 4 doi: 10.1016/j.ceca.2016.03.003. PMID: 26996063
Wang YX, Le WD
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Santa KM
Pharmacotherapy 2010 Nov;30(11):1179-96. doi: 10.1592/phco.30.11.1179. PMID: 20973690
Poulton J, Marchington DR
Neuromuscul Disord 2000 Oct;10(7):484-7. doi: 10.1016/s0960-8966(00)00130-9. PMID: 10996778

Clinical prediction guides

Finsterer J
Expert Opin Pharmacother 2019 Jul;20(10):1289-1297. Epub 2019 May 7 doi: 10.1080/14656566.2019.1609941. PMID: 31063406
El-Hattab AW, Adesina AM, Jones J, Scaglia F
Mol Genet Metab 2015 Sep-Oct;116(1-2):4-12. Epub 2015 Jun 15 doi: 10.1016/j.ymgme.2015.06.004. PMID: 26095523
Poulton J, Marchington DR
Neuromuscul Disord 2000 Oct;10(7):484-7. doi: 10.1016/s0960-8966(00)00130-9. PMID: 10996778
Santorelli FM, Siciliano G, Casali C, Basirico MG, Carrozzo R, Calvosa F, Sartucci F, Bonfiglio L, Murri L, DiMauro S
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Poulton J
Br J Hosp Med 1996 Jun 5-18;55(11):712-6. PMID: 8793140

Recent systematic reviews

Finsterer J
J Clin Neuromuscul Dis 2023 Mar 1;24(3):140-146. doi: 10.1097/CND.0000000000000422. PMID: 36809201

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