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Late-onset spinocerebellar degeneration

MedGen UID:
347273
Concept ID:
C1856604
Finding
HPO: HP:0006904

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVLate-onset spinocerebellar degeneration

Conditions with this feature

Gamma-glutamylcysteine synthetase deficiency
MedGen UID:
347272
Concept ID:
C1856603
Disease or Syndrome
Gamma-glutamylcysteine synthetase deficiency is 1 of 4 diseases involving enzymes in the gamma-glutamyl cycle (Meister, 1974). The other 3 disorders are glutathione synthetase deficiency (231900), 5-oxoprolinuria, which is a severe or generalized form of glutathione synthetase deficiency (266130), and gamma-glutamyl transpeptidase deficiency (231950). All except gamma-glutamyl transpeptidase deficiency are accompanied by hemolytic anemia (Larsson and Anderson, 2001). Some patients with CNSHA7 may develop progressive neurologic abnormalities, including ataxic gait, speech impairment, and spinocerebellar degeneration (Konrad et al., 1972). The hemolytic anemia caused by mutation in the GCLC gene is here designated congenital nonspherocytic hemolytic anemia-7 (CNSHA7).

Professional guidelines

PubMed

Leite Â, Dinis MA, Sequeiros J, Paúl C
Psychol Health Med 2017 Feb;22(2):244-249. Epub 2016 Mar 7 doi: 10.1080/13548506.2016.1159704. PMID: 26947204
Tanaka K, Sekijima Y, Yoshida K, Tamai M, Kosho T, Sakurai A, Wakui K, Ikeda S, Fukushima Y
J Hum Genet 2013 Aug;58(8):560-3. Epub 2013 May 2 doi: 10.1038/jhg.2013.34. PMID: 23635952
Brusse E, Maat-Kievit JA, van Swieten JC
Clin Genet 2007 Jan;71(1):12-24. doi: 10.1111/j.1399-0004.2006.00722.x. PMID: 17204042

Recent clinical studies

Etiology

Wirth T, Clément G, Delvallée C, Bonnet C, Bogdan T, Iosif A, Schalk A, Chanson JB, Pellerin D, Brais B, Roth V, Wandzel M, Fleury MC, Piton A, Calmels N, Namer IJ, Kremer S, Tranchant C, Renaud M, Anheim M
Mov Disord 2023 Oct;38(10):1950-1956. Epub 2023 Jul 20 doi: 10.1002/mds.29560. PMID: 37470282
Linares AJ, Fogel BL
Curr Opin Neurol 2023 Aug 1;36(4):324-334. Epub 2023 Jun 2 doi: 10.1097/WCO.0000000000001170. PMID: 37382141Free PMC Article
Wilke C, Pellerin D, Mengel D, Traschütz A, Danzi MC, Dicaire MJ, Neumann M, Lerche H, Bender B, Houlden H; RFC1 study group, Züchner S, Schöls L, Brais B, Synofzik M
Brain 2023 Oct 3;146(10):4144-4157. doi: 10.1093/brain/awad157. PMID: 37165652
Storey E
Semin Neurol 2014 Jul;34(3):280-92. Epub 2014 Sep 5 doi: 10.1055/s-0034-1386766. PMID: 25192506
Donato SD, Mariotti C, Taroni F
Handb Clin Neurol 2012;103:399-421. doi: 10.1016/B978-0-444-51892-7.00025-5. PMID: 21827903

Diagnosis

Pellerin D, Danzi MC, Renaud M, Houlden H, Synofzik M, Zuchner S, Brais B
Clin Transl Med 2024 Jan;14(1):e1504. doi: 10.1002/ctm2.1504. PMID: 38279833Free PMC Article
Linares AJ, Fogel BL
Curr Opin Neurol 2023 Aug 1;36(4):324-334. Epub 2023 Jun 2 doi: 10.1097/WCO.0000000000001170. PMID: 37382141Free PMC Article
Pellerin D, Danzi MC, Wilke C, Renaud M, Fazal S, Dicaire MJ, Scriba CK, Ashton C, Yanick C, Beijer D, Rebelo A, Rocca C, Jaunmuktane Z, Sonnen JA, Larivière R, Genís D, Molina Porcel L, Choquet K, Sakalla R, Provost S, Robertson R, Allard-Chamard X, Tétreault M, Reiling SJ, Nagy S, Nishadham V, Purushottam M, Vengalil S, Bardhan M, Nalini A, Chen Z, Mathieu J, Massie R, Chalk CH, Lafontaine AL, Evoy F, Rioux MF, Ragoussis J, Boycott KM, Dubé MP, Duquette A, Houlden H, Ravenscroft G, Laing NG, Lamont PJ, Saporta MA, Schüle R, Schöls L, La Piana R, Synofzik M, Zuchner S, Brais B
N Engl J Med 2023 Jan 12;388(2):128-141. Epub 2022 Dec 14 doi: 10.1056/NEJMoa2207406. PMID: 36516086Free PMC Article
Lieto M, Roca A, Santorelli FM, Fico T, De Michele G, Bellofatto M, Saccà F, De Michele G, Filla A
Neurol Sci 2019 Jul;40(7):1335-1342. Epub 2019 Mar 29 doi: 10.1007/s10072-019-03856-w. PMID: 30927137
Storey E
Semin Neurol 2014 Jul;34(3):280-92. Epub 2014 Sep 5 doi: 10.1055/s-0034-1386766. PMID: 25192506

Therapy

Abou Chaar W, Eranki AN, Stevens HA, Watson SL, Wong DY, Avila VS, Delfeld M, Gary AJ, Tawde S, Triebold M, Cherchi M, Xie T, Lockhart PJ, Bahlo M, Pellerin D, Dicaire MJ, Danzi M, Zuchner S, Brais BC, Perlman S, Burmeister M, Paulson H, Srinivasan S, Schut L, Bower M, Bushara K, Liao C, Shakkottai VG, Collins J, Clark HB, Das S, Fogel BL, Gomez CM
Ann Neurol 2024 Dec;96(6):1092-1103. Epub 2024 Sep 12 doi: 10.1002/ana.27060. PMID: 39263992Free PMC Article
Rummey C, Corben LA, Delatycki M, Wilmot G, Subramony SH, Corti M, Bushara K, Duquette A, Gomez C, Hoyle JC, Roxburgh R, Seeberger L, Yoon G, Mathews K, Zesiewicz T, Perlman S, Lynch DR
Neurology 2022 Oct 3;99(14):e1499-e1510. doi: 10.1212/WNL.0000000000200913. PMID: 35817567Free PMC Article
Reetz K, Dogan I, Hilgers RD, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesch S, Nachbauer W, Klopstock T, Stendel C, Rodríguez de Rivera Garrido FJ, Rummey C, Schöls L, Hayer SN, Klockgether T, Giordano I, Didszun C, Rai M, Pandolfo M, Schulz JB; EFACTS study group
Lancet Neurol 2021 May;20(5):362-372. Epub 2021 Mar 23 doi: 10.1016/S1474-4422(21)00027-2. PMID: 33770527
Koeppen AH, Becker AB, Feustel PJ, Gelman BB, Mazurkiewicz JE
J Neurol Sci 2016 Aug 15;367:171-6. Epub 2016 Jun 4 doi: 10.1016/j.jns.2016.06.006. PMID: 27423584
Keiser MS, Boudreau RL, Davidson BL
Mol Ther 2014 Mar;22(3):588-595. Epub 2013 Dec 12 doi: 10.1038/mt.2013.279. PMID: 24419082Free PMC Article

Prognosis

Ando M, Higuchi Y, Yuan J, Yoshimura A, Kojima F, Yamanishi Y, Aso Y, Izumi K, Imada M, Maki Y, Nakagawa H, Hobara T, Noguchi Y, Takei J, Hiramatsu Y, Nozuma S, Sakiyama Y, Hashiguchi A, Matsuura E, Okamoto Y, Takashima H
Ann Clin Transl Neurol 2024 Jan;11(1):96-104. Epub 2023 Nov 2 doi: 10.1002/acn3.51936. PMID: 37916889Free PMC Article
Koga S, Ali S, Baker MC, Wierenga KJ, Dompenciel M, Dickson DW, Wszolek ZK
Parkinsonism Relat Disord 2022 Dec;105:149-153. Epub 2022 Nov 11 doi: 10.1016/j.parkreldis.2022.11.008. PMID: 36396537
Palermo G, Mazzucchi S, Della Vecchia A, Siciliano G, Bonuccelli U, Azuar C, Ceravolo R, Lista S, Hampel H, Baldacci F
Mol Neurobiol 2020 Nov;57(11):4667-4691. Epub 2020 Aug 9 doi: 10.1007/s12035-020-02035-9. PMID: 32772223
Aguiar P, Pardo J, Arias M, Quintáns B, Fernández-Prieto M, Martínez-Regueiro R, Pumar JM, Silva-Rodríguez J, Ruibal Á, Sobrido MJ, Cortés J
Mov Disord 2017 Feb;32(2):264-273. Epub 2016 Nov 10 doi: 10.1002/mds.26854. PMID: 27862279
Brusse E, Maat-Kievit JA, van Swieten JC
Clin Genet 2007 Jan;71(1):12-24. doi: 10.1111/j.1399-0004.2006.00722.x. PMID: 17204042

Clinical prediction guides

Wirth T, Clément G, Delvallée C, Bonnet C, Bogdan T, Iosif A, Schalk A, Chanson JB, Pellerin D, Brais B, Roth V, Wandzel M, Fleury MC, Piton A, Calmels N, Namer IJ, Kremer S, Tranchant C, Renaud M, Anheim M
Mov Disord 2023 Oct;38(10):1950-1956. Epub 2023 Jul 20 doi: 10.1002/mds.29560. PMID: 37470282
Wilke C, Pellerin D, Mengel D, Traschütz A, Danzi MC, Dicaire MJ, Neumann M, Lerche H, Bender B, Houlden H; RFC1 study group, Züchner S, Schöls L, Brais B, Synofzik M
Brain 2023 Oct 3;146(10):4144-4157. doi: 10.1093/brain/awad157. PMID: 37165652
Reetz K, Dogan I, Hilgers RD, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesch S, Nachbauer W, Klopstock T, Stendel C, Rodríguez de Rivera Garrido FJ, Rummey C, Schöls L, Hayer SN, Klockgether T, Giordano I, Didszun C, Rai M, Pandolfo M, Schulz JB; EFACTS study group
Lancet Neurol 2021 May;20(5):362-372. Epub 2021 Mar 23 doi: 10.1016/S1474-4422(21)00027-2. PMID: 33770527
Palermo G, Mazzucchi S, Della Vecchia A, Siciliano G, Bonuccelli U, Azuar C, Ceravolo R, Lista S, Hampel H, Baldacci F
Mol Neurobiol 2020 Nov;57(11):4667-4691. Epub 2020 Aug 9 doi: 10.1007/s12035-020-02035-9. PMID: 32772223
Lecocq C, Charles P, Azulay JP, Meissner W, Rai M, N'Guyen K, Péréon Y, Fabre N, Robin E, Courtois S, Guyant-Maréchal L, Zagnoli F, Rudolf G, Renaud M, Sévin-Allouet M, Lesne F, Alaerts N, Goizet C, Calvas P, Eusebio A, Guissart C, Derkinderen P, Tison F, Brice A, Koenig M, Pandolfo M, Tranchant C, Dürr A, Anheim M
Mov Disord 2016 Jan;31(1):62-9. Epub 2015 Sep 21 doi: 10.1002/mds.26382. PMID: 26388117

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