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Page 1
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, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and d …
BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleuko …
Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy: Timing and Treatment.
Mallack EJ, Van Haren KP, Torrey A, van de Stadt S, Engelen M, Raymond GV, Fatemi A, Eichler FS. Mallack EJ, et al. Neurology. 2022 Aug 1;99(5):e512-e520. doi: 10.1212/WNL.0000000000200571. Neurology. 2022. PMID: 35609989 Free PMC article.
BACKGROUND AND OBJECTIVES: We sought to characterize the natural history and standard-of-care practices between the radiologic appearance of brain lesions, the appearance of lesional enhancement, and treatment with hematopoietic stem-cell transplant or gene therapy among boys dia …
BACKGROUND AND OBJECTIVES: We sought to characterize the natural history and standard-of-care practices between the radiologic appearance of …
Clinical and radiographic course of arrested cerebral adrenoleukodystrophy.
Mallack EJ, van de Stadt S, Caruso PA, Musolino PL, Sadjadi R, Engelen M, Eichler FS. Mallack EJ, et al. Neurology. 2020 Jun 16;94(24):e2499-e2507. doi: 10.1212/WNL.0000000000009626. Epub 2020 Jun 1. Neurology. 2020. PMID: 32482842 Free PMC article.
OBJECTIVE: To gain insight into the natural history of arrested cerebral adrenoleukodystrophy (CALD) by quantifying the change in Neurologic Function Score (NFS) and Loes Score (LS) over time in patients whose cerebral lesions spontaneously stopped pro …
OBJECTIVE: To gain insight into the natural history of arrested cerebral adrenoleukodystrophy (CALD) by quantifying the change …
Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies.
Ma CY, Li C, Zhou X, Zhang Z, Jiang H, Liu H, Chen HJ, Tse HF, Liao C, Lian Q. Ma CY, et al. Biomed Pharmacother. 2021 Nov;143:112214. doi: 10.1016/j.biopha.2021.112214. Epub 2021 Sep 21. Biomed Pharmacother. 2021. PMID: 34560537 Free article. Review.
Along with other factors such as epigenetic and environmental involvement, ABCD1 mutation-provoked disorders can present different phenotypes including cerebral adrenoleukodystrophy (cALD), adrenomyeloneuropathy (AMN), and peripheral neuropathy. cALD is the most sev …
Along with other factors such as epigenetic and environmental involvement, ABCD1 mutation-provoked disorders can present different phenotype …
Association between APOE4 and biomarkers in cerebral adrenoleukodystrophy.
Orchard PJ, Markowski TW, Higgins L, Raymond GV, Nascene DR, Miller WP, Pierpont EI, Lund TC. Orchard PJ, et al. Sci Rep. 2019 May 27;9(1):7858. doi: 10.1038/s41598-019-44140-3. Sci Rep. 2019. PMID: 31133696 Free PMC article.
Cerebral adrenoleukodystrophy (cALD) is an inflammatory neurodegenerative disease associated with mutation of the ABCD1 gene. ...In contrast, APOE4 carriers had an increased burden of cerebral disease involvement as determined by MRI severity score (10.5 vs 7 …
Cerebral adrenoleukodystrophy (cALD) is an inflammatory neurodegenerative disease associated with mutation of the ABCD1 gene. …
Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation.
Raymond GV, Aubourg P, Paker A, Escolar M, Fischer A, Blanche S, Baruchel A, Dalle JH, Michel G, Prasad V, Miller W, Paadre S, Balser J, Kurtzberg J, Nascene DR, Orchard PJ, Lund T. Raymond GV, et al. Biol Blood Marrow Transplant. 2019 Mar;25(3):538-548. doi: 10.1016/j.bbmt.2018.09.036. Epub 2018 Oct 4. Biol Blood Marrow Transplant. 2019. PMID: 30292747 Free article.
Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal neurodegenerative disease caused by mutations in the ABCD1 gene, resulting in deficiency of ALD protein. ...Neurologic function score (NFS) and Loes magnetic resonance imaging score …
Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal neurodegenerative disease caused by mutations in th …
Interval between contrast administration and T1-weighted MRI for cerebral adrenoleukodystrophy: a single-case observation.
Moscatelli M, Benzoni C, Doniselli FM, Verri M, Pascuzzo R, Aquino D, Mazzi F, Erbetta A, Salsano E. Moscatelli M, et al. Eur Radiol Exp. 2023 Oct 2;7(1):57. doi: 10.1186/s41747-023-00373-6. Eur Radiol Exp. 2023. PMID: 37782421 Free PMC article.
In ALD subjects, contrast-enhanced 2D T1-weighted gradient-echo sequences acquired at least 10 min after contrast injection may be considered to reduce false negative results.Relevance statementOur report is the first attempt to find an optimal delay between contrast administrati …
In ALD subjects, contrast-enhanced 2D T1-weighted gradient-echo sequences acquired at least 10 min after contrast injection may be considere …
Neurofilament light chain levels in cerebrospinal fluid as a sensitive biomarker for cerebral adrenoleukodystrophy.
Kakumoto T, Matsukawa T, Ishiura H, Mori H, Tsuji S, Toda T. Kakumoto T, et al. Ann Clin Transl Neurol. 2023 Jul;10(7):1230-1238. doi: 10.1002/acn3.51818. Epub 2023 May 31. Ann Clin Transl Neurol. 2023. PMID: 37259474 Free PMC article.
The correlation between cNfL levels and MRI-based Loes severity scores was investigated. A longitudinal analysis was performed on patients who underwent multiple CSF examinations. ...The cutoff cNfL level of 1930 pg/mL showed good sensitivity (95.5%) and specificity (85.7% …
The correlation between cNfL levels and MRI-based Loes severity scores was investigated. A longitudinal analysis was performed on pat …
Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.
Chiesa R, Boelens JJ, Duncan CN, Kühl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Chiesa R, et al. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294. Blood Adv. 2022. PMID: 34781360 Free PMC article.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic function and improve survival but has associated risks including transplant-related mortality (TRM), graft failure, and graft-versus-host d …
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic …
Childhood Cerebral Adrenoleukodystrophy: MR Perfusion Measurements and Their Use in Predicting Clinical Outcome after Hematopoietic Stem Cell Transplantation.
McKinney AM, Benson JC, Nascene DR, Eisengart J, Salmela MB, Loes DJ, Zhang L, Patel K, Raymond GV, Miller WP. McKinney AM, et al. AJNR Am J Neuroradiol. 2016 Sep;37(9):1713-20. doi: 10.3174/ajnr.A4773. Epub 2016 Apr 14. AJNR Am J Neuroradiol. 2016. PMID: 27079370 Free PMC article.
MR perfusion values were analyzed in the patients with cerebral adrenoleukodystrophy at each time point and compared with those in controls. ...CONCLUSIONS: Leading enhancing edge measurements likely remain normal post-hematopoietic stem cell transplantation in c …
MR perfusion values were analyzed in the patients with cerebral adrenoleukodystrophy at each time point and compared with thos …
50 results