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Select item 106592661.

A novel mouse model of cerebral adrenoleukodystrophy highlights NLRP3 activity in lesion pathogenesis

Ezzat Hashemi, Isha Narain Srivastava, Alejandro Aguirre, Ezra Tilahan Yoseph, Esha Kaushal, Avni Awani, Jae Kyu. Ryu, Katerina Akassoglou, Shahrzad Talebian, Pauline Chu, Laura Pisani, Patricia Musolino, Lawrence Steinman, Kristian Doyle, William H Robinson, Orr Sharpe, Romain Cayrol, Paul Orchard, Troy Lund, Hannes Vogel, Max Lenail, May Htwe Han, Joshua Leith Bonkowsky, Keith P. Van Haren

Version 1. bioRxiv. Preprint. 2023 Nov 10. doi: 10.1101/2023.11.07.564025

PMCID:: PMC10659266

Article PDF–2.4M

Select item 105456062.

Interval between contrast administration and T1-weighted MRI for cerebral adrenoleukodystrophy: a single-case observation

Marco Moscatelli, Chiara Benzoni, Fabio M. Doniselli, Mattia Verri, Riccardo Pascuzzo, Domenico Aquino, Federica Mazzi, Alessandra Erbetta, Ettore Salsano

Eur Radiol Exp. 2023 Dec; 7: 57. Published online 2023 Oct 2. doi: 10.1186/s41747-023-00373-6

PMCID:: PMC10545606

Article PDF–2.8M

Select item 104163833.

Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study

Caroline Sevin, Samira Hatteb, Aurore Clément, Fabrizia Bignami, Louis Chillotti, Françoise Bugnard, Stève Bénard, Odile Boespflug-Tanguy

Orphanet J Rare Dis. 2023; 18: 238. Published online 2023 Aug 10. doi: 10.1186/s13023-023-02843-x

PMCID:: PMC10416383

Article PDF–1.8M

Select item 103516524.

Neurofilament light chain levels in cerebrospinal fluid as a sensitive biomarker for cerebral adrenoleukodystrophy

Toshiyuki Kakumoto, Takashi Matsukawa, Hiroyuki Ishiura, Harushi Mori, Shoji Tsuji, Tatsushi Toda

Ann Clin Transl Neurol. 2023 Jul; 10(7): 1230–1238. Published online 2023 May 31. doi: 10.1002/acn3.51818

Correction in:: Ann Clin Transl Neurol. 2023 Sep; 10(9): 1700.

PMCID:: PMC10351652

Article PDF–1.2M

Select item 102892805.

Exploratory study of autophagy inducer sirolimus for childhood cerebral adrenoleukodystrophy

Xiao-Mei Luo, Li-Ying Liu, Qiu-Hong Wang, Yang-Yang Wang, Jing Wang, Xiao-Yan Yang, Shi-Jun Li, Li-Ping Zou

Front Pediatr. 2023; 11: 1187078. Published online 2023 Jun 6. doi: 10.3389/fped.2023.1187078

PMCID:: PMC10289280

Article PDF–2.3M

Select item 100762646.

Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy

Arne Lauer, Samantha L. Speroni, Myoung Choi, Xiao Da, Christine Duncan, Siobhan McCarthy, Vijai Krishnan, Cole A. Lusk, David Rohde, Mikkel Bo Hansen, Jayashree Kalpathy-Cramer, Daniel J. Loes, Paul A. Caruso, David A. Williams, Kim Mouridsen, Kyrre E. Emblem, Florian S. Eichler, Patricia L. Musolino

Nat Commun. 2023; 14: 1900. Published online 2023 Apr 5. doi: 10.1038/s41467-023-37262-w

PMCID:: PMC10076264

Article PDF–2.3M

Select item 95538437.

An in-silico approach to studying a very rare neurodegenerative disease using a disease with higher prevalence with shared pathways and genes: Cerebral adrenoleukodystrophy and Alzheimer’s disease

Yu Jeong Shim, Min Kyoung Shin, Junghyun Jung, Bongseong Koo, Wonhee Jang

Front Mol Neurosci. 2022; 15: 996698. Published online 2022 Sep 27. doi: 10.3389/fnmol.2022.996698

PMCID:: PMC9553843

Article PDF–4.7M

Select item 94216008.

Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment

Eric James Mallack, Keith P. Van Haren, Anna Torrey, Stephanie van de Stadt, Marc Engelen, Gerald V. Raymond, Ali Fatemi, Florian S. Eichler

Neurology. 2022 Aug 2; 99(5): e512–e520. doi: 10.1212/WNL.0000000000200571

Correction in:: Neurology. 2023 Jul 4; 101(1): 52.

PMCID:: PMC9421600

Article PDF–456K

Select item 91074559.

Glycoprotein nonmetastatic melanoma protein B (GNMPB) as a novel biomarker for cerebral adrenoleukodystrophy

Leyla A. Taghizadeh, Carina J. King, David R. Nascene, Ashish O. Gupta, Paul J. Orchard, LeeAnn Higgins, Todd W. Markowski, Erin E. Nolan, Justin W. Furcich, Troy C. Lund

Sci Rep. 2022; 12: 7985. Published online 2022 May 14. doi: 10.1038/s41598-022-11552-7

PMCID:: PMC9107455

Article PDF–1.8M

Select item 890939510.

Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral Adrenoleukodystrophy

Hongge Wang, Matthew D. Davison, Martin L. Kramer, Weiliang Qiu, Tatiana Gladysheva, Ruby M. S. Chiang, Can Kayatekin, David R. Nascene, Leyla A. Taghizadeh, Carina J. King, Erin E. Nolan, Ashish O. Gupta, Paul J. Orchard, Troy C. Lund

Cells. 2022 Mar; 11(5): 913. Published online 2022 Mar 7. doi: 10.3390/cells11050913

PMCID:: PMC8909395

Article PDF–965K

Select item 890569911.

Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy

Robert Chiesa, Jaap Jan Boelens, Christine N. Duncan, Jörn-Sven Kühl, Caroline Sevin, Neena Kapoor, Vinod K. Prasad, Caroline A. Lindemans, Simon A. Jones, Hernan M. Amartino, Mattia Algeri, Nancy Bunin, Cristina Diaz-de-Heredia, Daniel J. Loes, Esther Shamir, Alison Timm, Elizabeth McNeil, Andrew C. Dietz, Paul J. Orchard

Blood Adv. 2022 Mar 8; 6(5): 1512–1524. Published online 2022 Mar 3. doi: 10.1182/bloodadvances.2021005294

PMCID:: PMC8905699

Article PDF–1.3M

Select item 887451412.

Isoprostanoid Plasma Levels Are Relevant to Cerebral Adrenoleukodystrophy Disease

Cinzia Signorini, Claudio De Felice, Thierry Durand, Jean-Marie Galano, Camille Oger, Silvia Leoncini, Joussef Hayek, Jetty Chung-Yung Lee, Troy C. Lund, Paul J. Orchard

Life (Basel) 2022 Feb; 12(2): 146. Published online 2022 Jan 20. doi: 10.3390/life12020146

PMCID:: PMC8874514

Article PDF–3.3M

Select item 857839213.

Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy

Ashish O. Gupta, David R. Nascene, Ryan Shanley, Daniel L. Kenney‐Jung, Julie B. Eisengart, Troy C. Lund, Paul J. Orchard, Elizabeth I. Pierpont

J Inherit Metab Dis. 2021 Nov; 44(6): 1434–1440. Published online 2021 Sep 17. doi: 10.1002/jimd.12435

PMCID:: PMC8578392

Article PDF–1.4M

Select item 856483714.

ATYPICAL MRI FINDINGS IN CEREBRAL ADRENOLEUKODYSTROPHY: A CASE REPORT

Koray Koç, Arzu Canan, Pınar Koç, Kamil Karaali, Özgür Duman, Utku Şenol

Acta Clin Croat. 2021 Jun; 60(2): 326–328. doi: 10.20471/acc.2021.60.02.23

PMCID:: PMC8564837

Article PDF–320K

Select item 856273315.

A Longitudinal Analysis of Early Lesion Growth in Presymptomatic Patients with Cerebral Adrenoleukodystrophy

E.J. Mallack, G. Askin, S. van de Stadt, P.A. Caruso, P.L. Musolino, M. Engelen, S.N. Niogi, F.S. Eichler

AJNR Am J Neuroradiol. 2021 Oct; 42(10): 1904–1911. doi: 10.3174/ajnr.A7250

PMCID:: PMC8562733

Article PDF–1.9M

Select item 802568216.

Five men with arresting and relapsing cerebral adrenoleukodystrophy

Aaron M. Carlson, Irene C. Huffnagel, Aad Verrips, Marjo S. van der Knaap, Marc Engelen, Keith Van Haren

J Neurol. Author manuscript; available in PMC 2022 Mar 1.

Published in final edited form as: J Neurol. 2021 Mar; 268(3): 936–940. Published online 2020 Sep 29. doi: 10.1007/s00415-020-10225-7

PMCID:: PMC8025682

Article PDF–183K

Select item 749441217.

Adult-Onset Cerebral Adrenoleukodystrophy Without Adrenal Gland Involvement

Aniruddh Mannari, Brandon Wiggins, Ghassan Bachuwa

Cureus. 2020 Aug; 12(8): e9813. Published online 2020 Aug 17. doi: 10.7759/cureus.9813

PMCID:: PMC7494412

Article PDF–1.1M

Select item 745534918.

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

Elizabeth I. Pierpont, David R. Nascene, Ryan Shanley, Daniel L. Kenney-Jung, Richard S. Ziegler, Weston P. Miller, Ashish O. Gupta, Troy C. Lund, Paul J. Orchard, Julie B. Eisengart

Neurology. 2020 Aug 4; 95(5): e591–e600. doi: 10.1212/WNL.0000000000009929

PMCID:: PMC7455349

Article PDF–618K

Select item 745533819.

Clinical and radiographic course of arrested cerebral adrenoleukodystrophy

Eric J. Mallack, Stephanie van de Stadt, Paul A. Caruso, Patricia L. Musolino, Reza Sadjadi, Marc Engelen, Florian S. Eichler

Neurology. 2020 Jun 16; 94(24): e2499–e2507. doi: 10.1212/WNL.0000000000009626

PMCID:: PMC7455338

Article PDF–413K

Select item 741009520.

Rare Spontaneous Attenuation of Childhood Inflammatory Cerebral Adrenoleukodystrophy

Hyoung Won Choi, Gerald Vincent Raymond, Weston Miller

J Pediatr Neurol. Author manuscript; available in PMC 2020 Aug 6.

Published in final edited form as: J Pediatr Neurol. 2020 Apr; 18(2): 106–109. doi: 10.1055/s-0039-1677805

PMCID:: PMC7410095

Article PDF–396K

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