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Hereditary ataxia

MedGen UID:
2478
Concept ID:
C0004138
Disease or Syndrome
Synonym: Hereditary Ataxias
SNOMED CT: Hereditary ataxia (763597000)
 
Gene (location): KCND3 (1p13.2)
 
Monarch Initiative: MONDO:0100309
Orphanet: ORPHA183518

Definition

An instance of an atactic disorder that is caused by an inherited genomic modification in an individual. [from MONDO]

Professional guidelines

PubMed

de Silva R, Greenfield J, Cook A, Bonney H, Vallortigara J, Hunt B, Giunti P
Orphanet J Rare Dis 2019 Feb 20;14(1):51. doi: 10.1186/s13023-019-1013-9. PMID: 30786918Free PMC Article
Janssens AC
Arch Neurol 2005 Jan;62(1):164; author reply 164. doi: 10.1001/archneur.62.1.164-a. PMID: 15642867
Yakura H, Wakisaka A, Fujimoto S, Itakura K
N Engl J Med 1974 Jul 18;291(3):154-5. doi: 10.1056/NEJM197407182910314. PMID: 4833925

Recent clinical studies

Etiology

Morsy H, Benkirane M, Cali E, Rocca C, Zhelcheska K, Cipriani V, Galanaki E, Maroofian R, Efthymiou S, Murphy D, O'Driscoll M, Suri M, Banka S, Clayton-Smith J, Wright T, Redman M, Bassetti JA, Nizon M, Cogne B, Jamra RA, Bartolomaeus T, Heruth M, Krey I, Gburek-Augustat J, Wieczorek D, Gattermann F, Mcentagart M, Goldenberg A, Guyant-Marechal L, Garcia-Moreno H, Giunti P, Chabrol B, Bacrot S, Buissonnière R, Magry V, Gowda VK, Srinivasan VM, Melegh B, Szabó A, Sümegi K, Cossée M, Ziff M, Butterfield R, Hunt D, Bird-Lieberman G, Hanna M, Koenig M, Stankewich M, Vandrovcova J, Houlden H; Genomics England Research Consortium
Genet Med 2023 Jan;25(1):76-89. Epub 2022 Nov 4 doi: 10.1016/j.gim.2022.09.013. PMID: 36331550Free PMC Article
Galatolo D, De Michele G, Silvestri G, Leuzzi V, Casali C, Musumeci O, Antenora A, Astrea G, Barghigiani M, Battini R, Battisti C, Caputi C, Cioffi E, De Michele G, Dotti MT, Fico T, Fiorillo C, Galosi S, Lieto M, Malandrini A, Melone MAB, Mignarri A, Natale G, Pegoraro E, Petrucci A, Ricca I, Riso V, Rossi S, Rubegni A, Scarlatti A, Tinelli F, Trovato R, Tedeschi G, Tessa A, Filla A, Santorelli FM
Int J Mol Sci 2021 Aug 6;22(16) doi: 10.3390/ijms22168490. PMID: 34445196Free PMC Article
Szpisjak L, Zadori D, Klivenyi P, Vecsei L
CNS Neurol Disord Drug Targets 2019;18(4):279-293. doi: 10.2174/1871527318666190311155846. PMID: 30864514
Ruano L, Melo C, Silva MC, Coutinho P
Neuroepidemiology 2014;42(3):174-83. Epub 2014 Mar 5 doi: 10.1159/000358801. PMID: 24603320
Alekseeva N, Kablinger AS, Pinkston J, Gonzalez-Toledo EC, Minagar A
Adv Neurol 2005;96:275-83. PMID: 16383226

Diagnosis

Pedroso JL, Vale TC, França Junior MC, Kauffman MA, Teive H, Barsottini OGP, Munhoz RP
Cerebellum 2022 Dec;21(6):1073-1084. Epub 2021 Nov 15 doi: 10.1007/s12311-021-01345-5. PMID: 34782953
Galatolo D, De Michele G, Silvestri G, Leuzzi V, Casali C, Musumeci O, Antenora A, Astrea G, Barghigiani M, Battini R, Battisti C, Caputi C, Cioffi E, De Michele G, Dotti MT, Fico T, Fiorillo C, Galosi S, Lieto M, Malandrini A, Melone MAB, Mignarri A, Natale G, Pegoraro E, Petrucci A, Ricca I, Riso V, Rossi S, Rubegni A, Scarlatti A, Tinelli F, Trovato R, Tedeschi G, Tessa A, Filla A, Santorelli FM
Int J Mol Sci 2021 Aug 6;22(16) doi: 10.3390/ijms22168490. PMID: 34445196Free 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
Ashizawa T, Xia G
Continuum (Minneap Minn) 2016 Aug;22(4 Movement Disorders):1208-26. doi: 10.1212/CON.0000000000000362. PMID: 27495205Free PMC Article
Ruano L, Melo C, Silva MC, Coutinho P
Neuroepidemiology 2014;42(3):174-83. Epub 2014 Mar 5 doi: 10.1159/000358801. PMID: 24603320

Therapy

Liu Y, Ma Y, Zhang J, Yan X, Ouyang Y
Cerebellum 2024 Aug;23(4):1614-1625. Epub 2023 Nov 29 doi: 10.1007/s12311-023-01638-x. PMID: 38019418
Nanetti L, Alpini D, Mattei V, Castaldo A, Mongelli A, Brenna G, Gellera C, Mariotti C
Gait Posture 2017 Sep;57:11-14. Epub 2017 May 17 doi: 10.1016/j.gaitpost.2017.05.007. PMID: 28551466
Vogel AP, Keage MJ, Johansson K, Schalling E
Cochrane Database Syst Rev 2015 Nov 13;2015(11):CD010169. doi: 10.1002/14651858.CD010169.pub2. PMID: 26564018Free PMC Article
Vogel AP, Folker J, Poole ML
Cochrane Database Syst Rev 2014 Oct 28;2014(10):CD008953. doi: 10.1002/14651858.CD008953.pub2. PMID: 25348587Free PMC Article
Tomassini B, Arcuri G, Fortuni S, Sandi C, Ezzatizadeh V, Casali C, Condò I, Malisan F, Al-Mahdawi S, Pook M, Testi R
Hum Mol Genet 2012 Jul 1;21(13):2855-61. Epub 2012 Mar 23 doi: 10.1093/hmg/dds110. PMID: 22447512Free PMC Article

Prognosis

Witek N, Hawkins J, Hall D
Curr Neurol Neurosci Rep 2021 Feb 26;21(3):13. doi: 10.1007/s11910-021-01092-4. PMID: 33638050
Nanetti L, Alpini D, Mattei V, Castaldo A, Mongelli A, Brenna G, Gellera C, Mariotti C
Gait Posture 2017 Sep;57:11-14. Epub 2017 May 17 doi: 10.1016/j.gaitpost.2017.05.007. PMID: 28551466
Vogel AP, Wardrop MI, Folker JE, Synofzik M, Corben LA, Delatycki MB, Awan SN
J Voice 2017 Mar;31(2):243.e9-243.e19. Epub 2016 Aug 5 doi: 10.1016/j.jvoice.2016.04.015. PMID: 27501923
Alekseeva N, Kablinger AS, Pinkston J, Gonzalez-Toledo EC, Minagar A
Adv Neurol 2005;96:275-83. PMID: 16383226
Nance MA, Sevenich EA, Schut LJ
Am J Med Genet 1994 Sep 15;54(3):242-8. doi: 10.1002/ajmg.1320540310. PMID: 7810581

Clinical prediction guides

Pérez-Luz S, Gimenez-Cassina A, Fernández-Frías I, Wade-Martins R, Díaz-Nido J
Genomics 2015 Aug;106(2):76-82. Epub 2015 May 28 doi: 10.1016/j.ygeno.2015.05.006. PMID: 26027909
Vogel AP, Folker J, Poole ML
Cochrane Database Syst Rev 2014 Oct 28;2014(10):CD008953. doi: 10.1002/14651858.CD008953.pub2. PMID: 25348587Free PMC Article
Alekseeva N, Kablinger AS, Pinkston J, Gonzalez-Toledo EC, Minagar A
Adv Neurol 2005;96:275-83. PMID: 16383226
Pianese L, Busino L, De Biase I, De Cristofaro T, Lo Casale MS, Giuliano P, Monticelli A, Turano M, Criscuolo C, Filla A, Varrone S, Cocozza S
Hum Mol Genet 2002 Nov 1;11(23):2989-96. doi: 10.1093/hmg/11.23.2989. PMID: 12393810
Nance MA, Sevenich EA, Schut LJ
Am J Med Genet 1994 Sep 15;54(3):242-8. doi: 10.1002/ajmg.1320540310. PMID: 7810581

Recent systematic reviews

Pallarès-Sastre M, García M, Rouco-Axpe I, Amayra I
Brain Res 2024 Mar 15;1827:148765. Epub 2024 Jan 14 doi: 10.1016/j.brainres.2024.148765. PMID: 38219813
Liu Y, Ma Y, Zhang J, Yan X, Ouyang Y
Cerebellum 2024 Aug;23(4):1614-1625. Epub 2023 Nov 29 doi: 10.1007/s12311-023-01638-x. PMID: 38019418
Winser S, Chan HK, Chen WK, Hau CY, Leung SH, Leung YH, Bello UM
Physiother Theory Pract 2023 Jul 3;39(7):1355-1375. Epub 2022 Feb 25 doi: 10.1080/09593985.2022.2037115. PMID: 35212247
Vogel AP, Folker J, Poole ML
Cochrane Database Syst Rev 2014 Oct 28;2014(10):CD008953. doi: 10.1002/14651858.CD008953.pub2. PMID: 25348587Free PMC Article
Ruano L, Melo C, Silva MC, Coutinho P
Neuroepidemiology 2014;42(3):174-83. Epub 2014 Mar 5 doi: 10.1159/000358801. PMID: 24603320

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