ArticleEndocrinology2017
A Thyroid Hormone-Based Strategy for Correcting the Biochemical Abnormality in X-Linked Adrenoleukodystrophy.
Article in Endocrinology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
24 citing papers in PubMed, 68 citations in OpenAlex.
- Emerging Roles, Mechanisms, and Therapeutic Potential of Thyroid Hormones in Neurodegenerative Diseases: A Review.Brain sciences · 2026Review
- Review
- Drug discovery targeting thyroid hormone receptorActa pharmaceutica Sinica. B · 2025Review
- Selective Agonists of Thyroid Hormone Receptor Beta: Promising Tools for the Treatment of Nonalcoholic Fatty Liver Disease.Endocrinology and metabolism (Seoul, Korea) · 2024Article
- Targeted Brain Delivery of Dendrimer-4-Phenylbutyrate Ameliorates Neurological Deficits in a Long-Term ABCD1-Deficient Mouse Model of X-Linked Adrenoleukodystrophy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023Article
- Beneficial Effects of the Direct AMP-Kinase Activator PXL770 in In Vitro and In Vivo Models of X-Linked Adrenoleukodystrophy.The Journal of pharmacology and experimental therapeutics · 2022Article
- Drug discovery for X-linked adrenoleukodystrophy: An unbiased screen for compounds that lower very long-chain fatty acids.Journal of cellular biochemistry · 2021Article
- Peroxisomal ABC Transporters: An Update.International journal of molecular sciences · 2021Review
- Rare Diseases of Larynx, Trachea and Thyroid.Laryngo- rhino- otologie · 2021Review
- ABCD1 gene mutation in an Italian family with X-linkedadrenoleukodystrophy: case series.Endocrinology, diabetes & metabolism case reports · 2021Article
- Thyroid Hormone Analogues: An Update.Thyroid : official journal of the American Thyroid Association · 2020Review
- Pharmacological Complementation Remedies an Inborn Error of Lipid Metabolism.Cell chemical biology · 2020Article
- X-linked adrenoleukodystrophy: Pathology, pathophysiology, diagnostic testing, newborn screening and therapies.International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience · 2020Review
- MCT8 Deficiency: The Road to Therapies for a Rare Disease.Frontiers in neuroscience · 2020Review
- Selective Thyroid Hormone Receptor-Beta (TRβ) Agonists: New Perspectives for the Treatment of Metabolic and Neurodegenerative Disorders.Frontiers in medicine · 2020Review
- Myelin repair stimulated by CNS-selective thyroid hormone action.JCI insight · 2019Article
- X-LINKED ADRENOLEUKODYSTROPHY IN BRAZIL: A CASE SERIES.Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao Paulo · 2019Article
- Sobetirome and its Amide Prodrug Sob-AM2 Exert Thyromimetic Actions in Mct8-Deficient Brain.Thyroid : official journal of the American Thyroid Association · 2018Article
- Hypothalamic-Pituitary-Thyroid Axis Perturbations in Male Mice by CNS-Penetrating Thyromimetics.Endocrinology · 2018Article
- Thyroid hormone and the brain: Mechanisms of action in development and role in protection and promotion of recovery after brain injury.Pharmacology & therapeutics · 2018Review
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
X-linked adrenoleukodystrophy (X-ALD) is a rare, genetic disorder characterized by adrenal insufficiency and central nervous system (CNS) demyelination. All patients with X-ALD have the biochemical abnormality of elevated blood and tissue levels of very long chain fatty acids (VLCFAs), saturated fatty acids with 24 to 26 carbons. X-ALD results from loss of function mutations in the gene encoding the peroxisomal transporter ABCD1, which is responsible for uptake of VLCFAs into peroxisomes for degradation by oxidation. One proposed therapeutic strategy for genetic complementation of ABCD1 is pharmacologic upregulation of ABCD2, a gene encoding a homologous peroxisomal transporter. Here, we show that thyroid hormone or sobetirome, a clinical-stage selective thyroid hormone receptor agonist, increases cerebral Abcd2 and lowers VLCFAs in blood, peripheral organs, and brains of mice with defective Abcd1. These results support an approach to treating X-ALD that involves a thyromimetic agent that reactivates VLCFA disposal both in the periphery and the CNS.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.