Adrenoleukodystrophy [NEW TOPIC PAGE]

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From PubMed Clinical Queries

PubMed Clinical Queries is an utility offered by PubMed to search scientific literature in specific clinical research areas

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• Search PubMed Clinical Queries for "Adrenoleukodystrophy" 

Results: 5 of 306

• Clinical Trial of MGMT(P140K) Gene Therapy in the Treatment of Pediatric Patients with Brain Tumors.
  Kramer B, Singh R, Wischusen J, Dent R, Rush A, Middlemiss S, Ching YW, Alexander IE, McCowage G.Hum Gene Ther. 2018 Mar 23; . Epub 2018 Mar 23.
• Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
  Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, et al.N Engl J Med. 2017 Oct 26; 377(17):1630-1638. Epub 2017 Oct 4.
• [Novel therapies in neurometabolic diseases: the importance of early intervention].
  Gutierrez-Solana LG.Rev Neurol. 2017 May 17; 64(s03):S25-S28.
• A first case of adrenomyeloneuropathy with mutation Y174S of the adrenoleukodystrophy gene.
  Horikawa Y, Enya M, Yoshikura N, Kitagawa J, Takashima S, Shimozawa N, Takeda J.Neuro Endocrinol Lett. 2017 Feb; 38(1):13-18.
• Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy.
  Kühl JS, Suarez F, Gillett GT, Hemmati PG, Snowden JA, Stadler M, Vuong GL, Aubourg P, Köhler W, Arnold R.Brain. 2017 Apr 1; 140(4):953-966.

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This column displays citations filtered to a specific clinical study category and scope. These search filters were developed by Haynes RB et al. See more filter information.

Systematic Reviews

Results: 5 of 12

• Newborn screening for X-linked adrenoleukodystrophy in New York State: diagnostic protocol, surveillance protocol and treatment guidelines.
  Vogel BH, Bradley SE, Adams DJ, D'Aco K, Erbe RW, Fong C, Iglesias A, Kronn D, Levy P, Morrissey M, et al.Mol Genet Metab. 2015 Apr; 114(4):599-603. Epub 2015 Feb 12.
• Gene therapy as a new treatment option for inherited monogenic diseases.
  Boudes PF.Eur J Intern Med. 2014 Jan; 25(1):31-6. Epub 2013 Oct 12.
• Genotoxicity of retroviral hematopoietic stem cell gene therapy.
  Trobridge GD.Expert Opin Biol Ther. 2011 May; 11(5):581-93. Epub 2011 Mar 7.
• Graves' orbitopathy in a patient with adrenoleukodystrophy after bone marrow transplantation.
  Vardizer Y, Lupetti A, Vandelanotte S, Lankester AC, Wiersinga WM, Baldeschi L.Eur J Endocrinol. 2009 Aug; 161(2):369-73. Epub 2009 May 18.
• [Our experience in the diagnosis of peroxisomal diseases with an abnormal fatty acid profile].
  López-Pisón J, Pérez-Delgado R, García-Oguiza A, Lafuente-Hidalgo M, García-Jiménez M, Calvo-Ruata ML, Peña-Segura JL, Rebage V, Girós-Blasco M, Coll MJ, et al.Rev Neurol. 2008 Jul 1-15; 47(1):1-5.

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This column displays citations for systematic reviews, meta-analyses, reviews of clinical trials, evidence-based medicine, consensus development conferences, and guidelines. See filter information or additional related sources.

Medical Genetics

Topic:AllDiagnosisDifferential DiagnosisClinical DescriptionManagementGenetic CounselingMolecular GeneticsGenetic Testing

Results: 5 of 1075

• Impaired plasticity of macrophages in X-linked adrenoleukodystrophy.
  Weinhofer I, Zierfuss B, Hametner S, Wagner M, Popitsch N, Machacek C, Bartolini B, Zlabinger G, Ohradanova-Repic A, Stockinger H, et al.Brain. 2018 May 30; . Epub 2018 May 30.
• A novel mutation in the ABCD1 gene of a Chinese patient with X-linked adrenoleukodystrophy: Case report.
  Wang J, Zhu Q, Liu H.Medicine (Baltimore). 2018 May; 97(21):e10837.
• Etiology and treatment of adrenoleukodystrophy: new insights from <i>Drosophila</i>.
  Gordon HB, Valdez L, Letsou A.Dis Model Mech. 2018 Jun 15; 11(6). Epub 2018 Jun 15.
• Modeling and rescue of defective blood-brain barrier function of induced brain microvascular endothelial cells from childhood cerebral adrenoleukodystrophy patients.
  Lee CAA, Seo HS, Armien AG, Bates FS, Tolar J, Azarin SM.Fluids Barriers CNS. 2018 Apr 4; 15(1):9. Epub 2018 Apr 4.
• [Overview of X-linked adrenoleukodystrophy in Morocco: results of the implementation of the program of clinical and biological diagnosis].
  Benjelloun FM, Chabraoui L, Kriouile Y.Pan Afr Med J. 2017; 28:185. Epub 2017 Oct 30.

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This column displays citations pertaining to topics in medical genetics. See more filter information.