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Lactic Acidosis with Ataxia, Type I

MedGen UID:
199690
Concept ID:
C0751597
Disease or Syndrome
Synonyms: Ataxia with Lactic Acidosis; Ataxia with Lactic Acidosis I; Ataxia with Lactic Acidosis, Type I; Type I Ataxia with Lactic Acidosis
 
OMIM®: 312170

Professional guidelines

PubMed

Karissa P, Simpson T, Dawson SP, Low TY, Tay SH, Nordin FDA, Zain SM, Lee PY, Pung YF
Br J Biomed Sci 2022;79:10382. Epub 2022 May 19 doi: 10.3389/bjbs.2022.10382. PMID: 35996497Free PMC Article
Wasim M, Awan FR, Khan HN, Tawab A, Iqbal M, Ayesha H
Biochem Genet 2018 Apr;56(1-2):7-21. Epub 2017 Nov 1 doi: 10.1007/s10528-017-9825-6. PMID: 29094226
Stacpoole PW, Kurtz TL, Han Z, Langaee T
Adv Drug Deliv Rev 2008 Oct-Nov;60(13-14):1478-87. Epub 2008 Jul 4 doi: 10.1016/j.addr.2008.02.014. PMID: 18647626Free PMC Article

Recent clinical studies

Etiology

Zweers H, van Wegberg AMJ, Janssen MCH, Wortmann SB
Orphanet J Rare Dis 2021 Jul 3;16(1):295. doi: 10.1186/s13023-021-01927-w. PMID: 34217336Free PMC Article
Prasad N, Gottlich C, Nhan D, Hamosh A, Sponseller PD
J Pediatr Orthop 2021 Jul 1;41(6):e457-e463. doi: 10.1097/BPO.0000000000001812. PMID: 34096551
Wasim M, Awan FR, Khan HN, Tawab A, Iqbal M, Ayesha H
Biochem Genet 2018 Apr;56(1-2):7-21. Epub 2017 Nov 1 doi: 10.1007/s10528-017-9825-6. PMID: 29094226
Bird S
Aust Fam Physician 2014 May;43(5):331-2. PMID: 24791779
Robinson BH, Sherwood WG
J Inherit Metab Dis 1984;7 Suppl 1:69-73. doi: 10.1007/BF03047378. PMID: 6434848

Diagnosis

Zweers H, van Wegberg AMJ, Janssen MCH, Wortmann SB
Orphanet J Rare Dis 2021 Jul 3;16(1):295. doi: 10.1186/s13023-021-01927-w. PMID: 34217336Free PMC Article
Wasim M, Awan FR, Khan HN, Tawab A, Iqbal M, Ayesha H
Biochem Genet 2018 Apr;56(1-2):7-21. Epub 2017 Nov 1 doi: 10.1007/s10528-017-9825-6. PMID: 29094226
Chen Z, Zhong C
Prog Neurobiol 2013 Sep;108:21-43. Epub 2013 Jul 11 doi: 10.1016/j.pneurobio.2013.06.004. PMID: 23850509
De Meirleir L
Handb Clin Neurol 2013;113:1667-73. doi: 10.1016/B978-0-444-59565-2.00034-4. PMID: 23622387
Robinson BH, Sherwood WG
J Inherit Metab Dis 1984;7 Suppl 1:69-73. doi: 10.1007/BF03047378. PMID: 6434848

Therapy

Karissa P, Simpson T, Dawson SP, Low TY, Tay SH, Nordin FDA, Zain SM, Lee PY, Pung YF
Br J Biomed Sci 2022;79:10382. Epub 2022 May 19 doi: 10.3389/bjbs.2022.10382. PMID: 35996497Free PMC Article
Lavorato M, Nakamaru-Ogiso E, Mathew ND, Herman E, Shah N, Haroon S, Xiao R, Seiler C, Falk MJ
JCI Insight 2022 Aug 22;7(16) doi: 10.1172/jci.insight.156346. PMID: 35881484Free PMC Article
Zweers H, van Wegberg AMJ, Janssen MCH, Wortmann SB
Orphanet J Rare Dis 2021 Jul 3;16(1):295. doi: 10.1186/s13023-021-01927-w. PMID: 34217336Free PMC Article
Pasca L, De Giorgis V, Macasaet JA, Trentani C, Tagliabue A, Veggiotti P
Eur J Pediatr 2016 Oct;175(10):1267-76. Epub 2016 Sep 1 doi: 10.1007/s00431-016-2765-z. PMID: 27586246
Brown G
J Inherit Metab Dis 2014 Jul;37(4):577-85. Epub 2014 May 1 doi: 10.1007/s10545-014-9712-9. PMID: 24789339

Prognosis

Verma A, Lehman AN, Gokcan H, Cropcho L, Black D, Dobrowolski SF, Vockley J, Bedoyan JK
Mol Genet Genomic Med 2024 Jan;12(1):e2283. Epub 2023 Sep 8 doi: 10.1002/mgg3.2283. PMID: 37688338Free PMC Article
Zweers H, van Wegberg AMJ, Janssen MCH, Wortmann SB
Orphanet J Rare Dis 2021 Jul 3;16(1):295. doi: 10.1186/s13023-021-01927-w. PMID: 34217336Free PMC Article
Prasad N, Gottlich C, Nhan D, Hamosh A, Sponseller PD
J Pediatr Orthop 2021 Jul 1;41(6):e457-e463. doi: 10.1097/BPO.0000000000001812. PMID: 34096551
Patel KP, O'Brien TW, Subramony SH, Shuster J, Stacpoole PW
Mol Genet Metab 2012 Jan;105(1):34-43. Epub 2011 Oct 7 doi: 10.1016/j.ymgme.2011.09.032. PMID: 22079328Free PMC Article
Laurin JM, Lindor KD
Dig Dis 1994 Nov-Dec;12(6):331-50. doi: 10.1159/000171469. PMID: 7712616

Clinical prediction guides

Verma A, Lehman AN, Gokcan H, Cropcho L, Black D, Dobrowolski SF, Vockley J, Bedoyan JK
Mol Genet Genomic Med 2024 Jan;12(1):e2283. Epub 2023 Sep 8 doi: 10.1002/mgg3.2283. PMID: 37688338Free PMC Article
Gokcan H, Bedoyan JK, Isayev O
J Chem Inf Model 2022 Jul 25;62(14):3463-3475. Epub 2022 Jul 7 doi: 10.1021/acs.jcim.2c00630. PMID: 35797142Free PMC Article
Stacpoole PW, Kerr DS, Barnes C, Bunch ST, Carney PR, Fennell EM, Felitsyn NM, Gilmore RL, Greer M, Henderson GN, Hutson AD, Neiberger RE, O'Brien RG, Perkins LA, Quisling RG, Shroads AL, Shuster JJ, Silverstein JH, Theriaque DW, Valenstein E
Pediatrics 2006 May;117(5):1519-31. doi: 10.1542/peds.2005-1226. PMID: 16651305
Sugden MC, Holness MJ
Curr Drug Targets Immune Endocr Metabol Disord 2002 Jul;2(2):151-65. PMID: 12476789
Imahori K, Uchida T
J Biochem 1997 Feb;121(2):179-88. PMID: 9089387

Recent systematic reviews

Zweers H, van Wegberg AMJ, Janssen MCH, Wortmann SB
Orphanet J Rare Dis 2021 Jul 3;16(1):295. doi: 10.1186/s13023-021-01927-w. PMID: 34217336Free PMC Article

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