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Refsum disease, adult, 1

MedGen UID:
411814
Concept ID:
C2749345
Disease or Syndrome
Synonym: Refsum Disease, Adult, 1
 
OMIM®: 266500; 602026

Professional guidelines

PubMed

Smith AD, Refsum H, Bottiglieri T, Fenech M, Hooshmand B, McCaddon A, Miller JW, Rosenberg IH, Obeid R
J Alzheimers Dis 2018;62(2):561-570. doi: 10.3233/JAD-171042. PMID: 29480200Free PMC Article
DiGiovanna JJ, Robinson-Bostom L
Am J Clin Dermatol 2003;4(2):81-95. doi: 10.2165/00128071-200304020-00002. PMID: 12553849
Schutgens RB, Schrakamp G, Wanders RJ, Heymans HS, Tager JM, van den Bosch H
J Inherit Metab Dis 1989;12 Suppl 1:118-34. doi: 10.1007/BF01799291. PMID: 2509803

Recent clinical studies

Etiology

Wanders RJA, Baes M, Ribeiro D, Ferdinandusse S, Waterham HR
Physiol Rev 2023 Jan 1;103(1):957-1024. Epub 2022 Aug 11 doi: 10.1152/physrev.00051.2021. PMID: 35951481
Picher-Martel V, Dupre N
CNS Neurol Disord Drug Targets 2018;17(3):161-171. doi: 10.2174/1871527317666180419115029. PMID: 29676235
Pakzad-Vaezi KL, Maberley DA
Retin Cases Brief Rep 2014 Winter;8(1):56-9. doi: 10.1097/ICB.0000000000000004. PMID: 25372210
Aubourg P, Wanders R
Handb Clin Neurol 2013;113:1593-609. doi: 10.1016/B978-0-444-59565-2.00028-9. PMID: 23622381
Depreter M, Espeel M, Roels F
Microsc Res Tech 2003 Jun 1;61(2):203-23. doi: 10.1002/jemt.10330. PMID: 12740827

Diagnosis

Boltshauser E, Weber KP
Handb Clin Neurol 2018;154:287-298. doi: 10.1016/B978-0-444-63956-1.00017-5. PMID: 29903445
Pakzad-Vaezi KL, Maberley DA
Retin Cases Brief Rep 2014 Winter;8(1):56-9. doi: 10.1097/ICB.0000000000000004. PMID: 25372210
Aubourg P, Wanders R
Handb Clin Neurol 2013;113:1593-609. doi: 10.1016/B978-0-444-59565-2.00028-9. PMID: 23622381
Depreter M, Espeel M, Roels F
Microsc Res Tech 2003 Jun 1;61(2):203-23. doi: 10.1002/jemt.10330. PMID: 12740827
Aubourg P, Scotto J, Rocchiccioli F, Feldmann-Pautrat D, Robain O
J Neurol Neurosurg Psychiatry 1986 Jan;49(1):77-86. doi: 10.1136/jnnp.49.1.77. PMID: 2420940Free PMC Article

Therapy

Grande G, Hooshmand B, Vetrano DL, Smith DA, Refsum H, Fratiglioni L, Ljungman P, Wu J, Bellavia A, Eneroth K, Bellander T, Rizzuto D
Neurology 2023 Sep 19;101(12):e1231-e1240. Epub 2023 Jul 13 doi: 10.1212/WNL.0000000000207656. PMID: 37442622Free PMC Article
Picher-Martel V, Dupre N
CNS Neurol Disord Drug Targets 2018;17(3):161-171. doi: 10.2174/1871527317666180419115029. PMID: 29676235
Smith AD, Refsum H, Bottiglieri T, Fenech M, Hooshmand B, McCaddon A, Miller JW, Rosenberg IH, Obeid R
J Alzheimers Dis 2018;62(2):561-570. doi: 10.3233/JAD-171042. PMID: 29480200Free PMC Article
DiGiovanna JJ, Robinson-Bostom L
Am J Clin Dermatol 2003;4(2):81-95. doi: 10.2165/00128071-200304020-00002. PMID: 12553849
Ueland PM, Refsum H, Beresford SA, Vollset SE
Am J Clin Nutr 2000 Aug;72(2):324-32. doi: 10.1093/ajcn/72.2.324. PMID: 10919921

Prognosis

Yu J, Refsum E, Perrin V, Helsingen LM, Wieszczy P, Løberg M, Bretthauer M, Adami HO, Ye W, Blom J, Kalager M
Ann Oncol 2022 Jun;33(6):649-656. Epub 2022 Mar 8 doi: 10.1016/j.annonc.2022.02.226. PMID: 35276334
Arias M
Neurologia (Engl Ed) 2019 May;34(4):248-258. Epub 2016 Jul 25 doi: 10.1016/j.nrl.2016.06.006. PMID: 27460185
Palau F, Espinós C
Orphanet J Rare Dis 2006 Nov 17;1:47. doi: 10.1186/1750-1172-1-47. PMID: 17112370Free PMC Article
Depreter M, Espeel M, Roels F
Microsc Res Tech 2003 Jun 1;61(2):203-23. doi: 10.1002/jemt.10330. PMID: 12740827
Aubourg P, Scotto J, Rocchiccioli F, Feldmann-Pautrat D, Robain O
J Neurol Neurosurg Psychiatry 1986 Jan;49(1):77-86. doi: 10.1136/jnnp.49.1.77. PMID: 2420940Free PMC Article

Clinical prediction guides

Juul FE, Jodal HC, Barua I, Refsum E, Olsvik Ø, Helsingen LM, Løberg M, Bretthauer M, Kalager M, Emilsson L
Scand J Public Health 2022 Feb;50(1):38-45. Epub 2021 Oct 5 doi: 10.1177/14034948211047137. PMID: 34609261Free PMC Article
Olsen T, Vinknes KJ, Svingen GFT, Pedersen ER, Tell GS, Blomhoff R, Drevon CA, Ueland PM, Midttun Ø, Refsum H, Nygård OK
Atherosclerosis 2017 Oct;265:325-330. Epub 2017 Jul 21 doi: 10.1016/j.atherosclerosis.2017.07.020. PMID: 28774484
Dhawan A, Mitry RR, Hughes RD
J Inherit Metab Dis 2006 Apr-Jun;29(2-3):431-5. doi: 10.1007/s10545-006-0245-8. PMID: 16763914
Suzuki Y, Shimozawa N, Imamura A, Fukuda S, Zhang Z, Orii T, Kondo N
J Inherit Metab Dis 2001 Apr;24(2):151-65. doi: 10.1023/a:1010310816743. PMID: 11405337
Ueland PM, Refsum H, Beresford SA, Vollset SE
Am J Clin Nutr 2000 Aug;72(2):324-32. doi: 10.1093/ajcn/72.2.324. PMID: 10919921

Recent systematic reviews

Jennings MJ, Lochmüller A, Atalaia A, Horvath R
J Neuromuscul Dis 2021;8(3):383-400. doi: 10.3233/JND-200546. PMID: 32773395Free PMC Article

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