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Pelizaeus-Merzbacher disease, connatal

MedGen UID:
864920
Concept ID:
C4016483
Finding
Synonym: PELIZAEUS-MERZBACHER DISEASE, CONNATAL
 
OMIM®: 300401

Professional guidelines

PubMed

Biancheri R, Grossi S, Regis S, Rossi A, Corsolini F, Rossi DP, Cavalli P, Severino M, Filocamo M
Clin Genet 2014 Mar;85(3):267-72. Epub 2013 May 27 doi: 10.1111/cge.12154. PMID: 23711321

Recent clinical studies

Etiology

Ashrafi MR, Amanat M, Garshasbi M, Kameli R, Nilipour Y, Heidari M, Rezaei Z, Tavasoli AR
Expert Rev Neurother 2020 Jan;20(1):65-84. Epub 2019 Dec 12 doi: 10.1080/14737175.2020.1699060. PMID: 31829048
Lyahyai J, Ouled Amar Bencheikh B, Elalaoui SC, Mansouri M, Boualla L, DIonne-Laporte A, Spiegelman D, Dion PA, Cossette P, Rouleau GA, Sefiani A
BMC Pediatr 2018 Feb 27;18(1):90. doi: 10.1186/s12887-018-1063-5. PMID: 29486744Free PMC Article
Steenweg ME, Vanderver A, Blaser S, Bizzi A, de Koning TJ, Mancini GM, van Wieringen WN, Barkhof F, Wolf NI, van der Knaap MS
Brain 2010 Oct;133(10):2971-82. doi: 10.1093/brain/awq257. PMID: 20881161Free PMC Article
Svenstrup K, Giraud G, Boespflug-Tanguy O, Danielsen ER, Thomsen C, Rasmussen K, Law I, Vogel A, Stokholm J, Crone C, Hjermind LE, Nielsen JE
J Neurol Neurosurg Psychiatry 2010 Jun;81(6):666-72. Epub 2009 Dec 1 doi: 10.1136/jnnp.2009.180315. PMID: 19955111
Kuan CC, Sano M, Kaga K, Kodama M, Kodama K
Acta Otolaryngol 2008 May;128(5):539-46. doi: 10.1080/00016480701258713. PMID: 18421608

Diagnosis

Ashrafi MR, Amanat M, Garshasbi M, Kameli R, Nilipour Y, Heidari M, Rezaei Z, Tavasoli AR
Expert Rev Neurother 2020 Jan;20(1):65-84. Epub 2019 Dec 12 doi: 10.1080/14737175.2020.1699060. PMID: 31829048
Osório MJ, Goldman SA
Handb Clin Neurol 2018;148:701-722. doi: 10.1016/B978-0-444-64076-5.00045-4. PMID: 29478609Free PMC Article
Takanashi J
Magn Reson Med Sci 2015;14(2):85-91. doi: 10.2463/mrms.2014-0064. PMID: 26185828
Hobson GM, Garbern JY
Semin Neurol 2012 Feb;32(1):62-7. Epub 2012 Mar 15 doi: 10.1055/s-0032-1306388. PMID: 22422208
Woodward KJ
Expert Rev Mol Med 2008 May 19;10:e14. doi: 10.1017/S1462399408000677. PMID: 18485258

Therapy

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
Carrozzo R, Arrigo G, Rossi E, Bardoni B, Cammarata M, Gandullia P, Gatti R, Zuffardi O
Am J Med Genet 1997 Oct 31;72(3):329-34. PMID: 9332664

Prognosis

Perrier S, Gauquelin L, Bernard G
Handb Clin Neurol 2024;204:197-223. doi: 10.1016/B978-0-323-99209-1.00014-4. PMID: 39322379
Mierzewska H, Jamroz E, Mazurczak T, Hoffman-Zacharska D, Szczepanik E
Folia Neuropathol 2016;54(1):59-65. doi: 10.5114/fn.2016.58916. PMID: 27179222
Numata Y, Gotoh L, Iwaki A, Kurosawa K, Takanashi J, Deguchi K, Yamamoto T, Osaka H, Inoue K
J Neurol 2014 Apr;261(4):752-8. Epub 2014 Feb 16 doi: 10.1007/s00415-014-7263-5. PMID: 24532200
Coker SB, Thomas C
Clin Pediatr (Phila) 1995 Jul;34(7):349-52. doi: 10.1177/000992289503400702. PMID: 7554683
Scheffer IE, Baraitser M, Wilson J, Harding B, Kendall B, Brett EM
Neuropediatrics 1991 May;22(2):71-8. doi: 10.1055/s-2008-1071420. PMID: 1857497

Clinical prediction guides

Harting I, Garbade SF, Rosendaal SD, Mohr A, Sherbini O, Vanderver A, Wolf NI
Eur J Paediatr Neurol 2022 Nov;41:71-79. Epub 2022 Nov 4 doi: 10.1016/j.ejpn.2022.10.003. PMID: 36368233Free PMC Article
Numata Y, Gotoh L, Iwaki A, Kurosawa K, Takanashi J, Deguchi K, Yamamoto T, Osaka H, Inoue K
J Neurol 2014 Apr;261(4):752-8. Epub 2014 Feb 16 doi: 10.1007/s00415-014-7263-5. PMID: 24532200
Biancheri R, Grossi S, Regis S, Rossi A, Corsolini F, Rossi DP, Cavalli P, Severino M, Filocamo M
Clin Genet 2014 Mar;85(3):267-72. Epub 2013 May 27 doi: 10.1111/cge.12154. PMID: 23711321
Mayer JA, Larsen EC, Kondo Y, Duncan ID
Neurobiol Dis 2011 Nov;44(2):231-8. Epub 2011 Jul 18 doi: 10.1016/j.nbd.2011.07.007. PMID: 21784154
Gencic S, Abuelo D, Ambler M, Hudson LD
Am J Hum Genet 1989 Sep;45(3):435-42. PMID: 2773936Free PMC Article

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