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Adrenoleukodystrophy

Serum very long-chain fatty acids (VLCFA) levels as predictive biomarkers of diseases severity and probability of survival in peroxisomal disorders

Teresa Joanna Stradomska, Małgorzata Syczewska, [ ... ], Anna Tylki-Szymanska

A combination of 7-ketocholesterol, lysosphingomyelin and bile acid-408 to diagnose Niemann-Pick disease type C using LC-MS/MS

Chen Wu, Takeo Iwamoto, [ ... ], Yoshikatsu Eto

Unmasking adrenoleukodystrophy in a cohort of cerebellar ataxia

Ying-Hao Chen, Yi-Chung Lee, [ ... ], Bing-Wen Soong

C26:0-Carnitine Is a New Biomarker for X-Linked Adrenoleukodystrophy in Mice and Man

Malu-Clair van de Beek, Inge M. E. Dijkstra, [ ... ], Stephan Kemp

Functional Characterization of IPSC-Derived Brain Cells as a Model for X-Linked Adrenoleukodystrophy

Mauhamad Baarine, Mushfiquddin Khan, Avtar Singh, Inderjit Singh

Abcd2 Is a Strong Modifier of the Metabolic Impairments in Peritoneal Macrophages of Abcd1-Deficient Mice

Zahid Muneer, Christoph Wiesinger, [ ... ], Sonja Forss-Petter

Evaluation of Retinoids for Induction of the Redundant Gene ABCD2 as an Alternative Treatment Option in X-Linked Adrenoleukodystrophy

Franziska D. Weber, Isabelle Weinhofer, [ ... ], Johannes Berger

Histone Deacetylase Inhibitor Upregulates Peroxisomal Fatty Acid Oxidation and Inhibits Apoptotic Cell Death in Abcd1-Deficient Glial Cells

Jaspreet Singh, Mushfiquddin Khan, [ ... ], Inderjit Singh

Familial Skewed X Chromosome Inactivation in Adrenoleukodystrophy Manifesting Heterozygotes from a Chinese Pedigree

Zhihong Wang, Aizhen Yan, [ ... ], Fenghua Lan

ABCD2 Is a Direct Target of β-Catenin and TCF-4: Implications for X-Linked Adrenoleukodystrophy Therapy

Chul-Yong Park, Han-Soo Kim, [ ... ], Dong-Wook Kim

X-Linked Adrenoleukodystrophy: Molecular and Functional Analysis of the ABCD1 Gene in Argentinean Patients

Cyntia Anabel Amorosi, Helena Myskóva, [ ... ], Ana María Oller de Ramírez

Cerebrospinal Fluid Matrix Metalloproteinases Are Elevated in Cerebral Adrenoleukodystrophy and Correlate with MRI Severity and Neurologic Dysfunction

Kathryn A. Thibert, Gerald V. Raymond, [ ... ], Troy C. Lund

Bezafibrate for X-Linked Adrenoleukodystrophy

Marc Engelen, Luc Tran, [ ... ], Stephan Kemp

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