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Hereditary Central Nervous System Demyelinating Diseases

MedGen UID:
148407
Concept ID:
C0751877
Disease or Syndrome
Synonyms: Central Nervous System Demyelinating Diseases, Hereditary; Central Nervous System Demyelinating Hereditary Diseases; Central Nervous System Hereditary Demyelinating Diseases; Demyelinating Central Nervous System Diseases, Hereditary; Demyelinating Diseases, Central Nervous System, Hereditary; Hereditary Demyelinating Diseases, Central Nervous System

Definition

Inherited conditions characterized by a loss of MYELIN in the central nervous system. [from MeSH]

Professional guidelines

PubMed

Gujral J, Sethuram S
Curr Opin Endocrinol Diabetes Obes 2023 Feb 1;30(1):44-51. Epub 2022 Nov 14 doi: 10.1097/MED.0000000000000782. PMID: 36373727
Turk BR, Theda C, Fatemi A, Moser AB
Int J Dev Neurosci 2020 Feb;80(1):52-72. Epub 2020 Jan 26 doi: 10.1002/jdn.10003. PMID: 31909500Free PMC Article
Engelen M, Kemp S, Poll-The BT
Curr Neurol Neurosci Rep 2014 Oct;14(10):486. doi: 10.1007/s11910-014-0486-0. PMID: 25115486

Recent clinical studies

Etiology

Ceravolo G, Zhelcheska K, Squadrito V, Pellerin D, Gitto E, Hartley L, Houlden H
J Neurol 2024 Jan;271(1):593-605. Epub 2023 Sep 27 doi: 10.1007/s00415-023-11996-5. PMID: 37755460Free PMC Article
Sarret C
Rev Neurol (Paris) 2020 Jan-Feb;176(1-2):10-19. Epub 2019 Jun 4 doi: 10.1016/j.neurol.2019.04.003. PMID: 31174885
Köhler W, Curiel J, Vanderver A
Nat Rev Neurol 2018 Feb;14(2):94-105. Epub 2018 Jan 5 doi: 10.1038/nrneurol.2017.175. PMID: 29302065Free PMC Article
Barañano KW
Semin Neurol 2016 Aug;36(4):362-6. Epub 2016 Sep 19 doi: 10.1055/s-0036-1585455. PMID: 27643905
Perlman SJ, Mar S
Adv Exp Med Biol 2012;724:154-71. doi: 10.1007/978-1-4614-0653-2_13. PMID: 22411242

Diagnosis

Sarret C
Rev Neurol (Paris) 2020 Jan-Feb;176(1-2):10-19. Epub 2019 Jun 4 doi: 10.1016/j.neurol.2019.04.003. PMID: 31174885
Resende LL, de Paiva ARB, Kok F, da Costa Leite C, Lucato LT
Radiographics 2019 Jan-Feb;39(1):153-168. doi: 10.1148/rg.2019180081. PMID: 30620693
Barañano KW
Semin Neurol 2016 Aug;36(4):362-6. Epub 2016 Sep 19 doi: 10.1055/s-0036-1585455. PMID: 27643905
Renaud DL
Semin Neurol 2012 Feb;32(1):3-8. Epub 2012 Mar 15 doi: 10.1055/s-0032-1306380. PMID: 22422201
Perlman SJ, Mar S
Adv Exp Med Biol 2012;724:154-71. doi: 10.1007/978-1-4614-0653-2_13. PMID: 22411242

Therapy

Hamanaka K, Miyatake S, Zerem A, Lev D, Blumkin L, Yokochi K, Fujita A, Imagawa E, Iwama K, Nakashima M, Mitsuhashi S, Mizuguchi T, Takata A, Miyake N, Saitsu H, van der Knaap MS, Lerman-Sagie T, Matsumoto N
J Hum Genet 2018 Dec;63(12):1223-1229. Epub 2018 Sep 27 doi: 10.1038/s10038-018-0516-x. PMID: 30258207
Tonduti D, Aiello C, Renaldo F, Dorboz I, Saaman S, Rodriguez D, Fettah H, Elmaleh M, Biancheri R, Barresi S, Boccone L, Orcesi S, Pichiecchio A, Zangaglia R, Maurey H, Rossi A, Boespflug-Tanguy O, Bertini E
Eur J Paediatr Neurol 2016 Mar;20(2):323-330. Epub 2015 Nov 28 doi: 10.1016/j.ejpn.2015.11.006. PMID: 26643067
Helman G, Van Haren K, Escolar ML, Vanderver A
Pediatr Clin North Am 2015 Jun;62(3):649-66. Epub 2015 Apr 8 doi: 10.1016/j.pcl.2015.03.006. PMID: 26022168Free PMC Article
Siva K, Covello G, Denti MA
Nucleic Acid Ther 2014 Feb;24(1):69-86. doi: 10.1089/nat.2013.0461. PMID: 24506781Free PMC Article
Jaklin C, Heiliger K, Hempel M, Sollacher D, Cohen M, Makowski CC, Meitinger T, Jauch A, Oexle K
Eur J Med Genet 2012 Oct;55(10):568-72. Epub 2012 Jul 7 doi: 10.1016/j.ejmg.2012.06.010. PMID: 22776853

Prognosis

Soderholm HE, Chapin AB, Bayrak-Toydemir P, Bonkowsky JL
Pediatr Neurol 2020 Oct;111:66-69. Epub 2020 Jun 17 doi: 10.1016/j.pediatrneurol.2020.06.005. PMID: 32951664Free PMC Article
Köhler W, Curiel J, Vanderver A
Nat Rev Neurol 2018 Feb;14(2):94-105. Epub 2018 Jan 5 doi: 10.1038/nrneurol.2017.175. PMID: 29302065Free PMC Article
Perlman SJ, Mar S
Adv Exp Med Biol 2012;724:154-71. doi: 10.1007/978-1-4614-0653-2_13. PMID: 22411242
Vanderver A
Curr Neurol Neurosci Rep 2005 Mar;5(2):110-8. doi: 10.1007/s11910-005-0008-1. PMID: 15743548
Barker PB, Horská A
J Child Neurol 2004 Aug;19(8):559-70. doi: 10.1177/088307380401900801. PMID: 15605464

Clinical prediction guides

Soderholm HE, Chapin AB, Bayrak-Toydemir P, Bonkowsky JL
Pediatr Neurol 2020 Oct;111:66-69. Epub 2020 Jun 17 doi: 10.1016/j.pediatrneurol.2020.06.005. PMID: 32951664Free PMC Article
Resende LL, de Paiva ARB, Kok F, da Costa Leite C, Lucato LT
Radiographics 2019 Jan-Feb;39(1):153-168. doi: 10.1148/rg.2019180081. PMID: 30620693
Mathis S, Scheper GC, Baumann N, Petit E, Gil R, van der Knaap MS, Neau JP
Clin Neurol Neurosurg 2008 Dec;110(10):1035-7. Epub 2008 Aug 3 doi: 10.1016/j.clineuro.2008.06.002. PMID: 18678442
Sedel F, Tourbah A, Fontaine B, Lubetzki C, Baumann N, Saudubray JM, Lyon-Caen O
J Inherit Metab Dis 2008 Jun;31(3):295-307. Epub 2008 Feb 25 doi: 10.1007/s10545-008-0778-0. PMID: 18344012
Fogli A, Boespflug-Tanguy O
Biochem Soc Trans 2006 Feb;34(Pt 1):22-9. doi: 10.1042/BST20060022. PMID: 16246171

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