Found: 22
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Biological Variation in Peripheral Inflammation and Oxidative Stress Biomarkers in Individuals with Gaucher Disease.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 16, p. 9189, doi. 10.3390/ijms23169189
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- Publication type:
- Article
Building cross-border collaborations to increase diversity and accelerate rare disease drug development -- meeting report from the inaugural IndoUSrare Annual Conference 2021.
- Published in:
- Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221133124
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- Publication type:
- Article
Combinatorial Treatment of DNA and Chromatin-Modifying Drugs Cause Cell Death in Human and Canine Osteosarcoma Cell Lines.
- Published in:
- PLoS ONE, 2012, v. 7, n. 9, p. 1, doi. 10.1371/journal.pone.0043720
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- Publication type:
- Article
The MiRNA Journey from Theory to Practice as a CNS Biomarker.
- Published in:
- Frontiers in Genetics, 2016, p. 1, doi. 10.3389/fgene.2016.00011
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- Publication type:
- Article
Translational and clinical pharmacology considerations in drug repurposing for X‐linked adrenoleukodystrophy—A rare peroxisomal disorder.
- Published in:
- British Journal of Clinical Pharmacology, 2022, v. 88, n. 6, p. 2552, doi. 10.1111/bcp.15090
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- Publication type:
- Article
A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy.
- Published in:
- British Journal of Clinical Pharmacology, 2016, v. 81, n. 6, p. 1058, doi. 10.1111/bcp.12897
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- Publication type:
- Article
Mechanisms of Antioxidant Induction with High-Dose N-Acetylcysteine in Childhood Cerebral Adrenoleukodystrophy.
- Published in:
- 2015
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- Publication type:
- journal article
Development of therapies for rare genetic disorders of GPX4: roadmap and opportunities.
- Published in:
- 2021
- By:
- Publication type:
- journal article
Intravital microscopy: An innovative approach to track real‐time muscle integrity and pathology in Pompe disease.
- Published in:
- Clinical & Translational Discovery, 2024, v. 4, n. 4, p. 1, doi. 10.1002/ctd2.322
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- Publication type:
- Article
N-Acetyl-L-cysteine Protects Human Retinal Pigment Epithelial Cells from Oxidative Damage: Implications for Age-Related Macular Degeneration.
- Published in:
- Oxidative Medicine & Cellular Longevity, 2019, p. 1, doi. 10.1155/2019/5174957
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- Publication type:
- Article
Physiologically‐Based Pharmacokinetic Model Development, Validation, and Application for Prediction of Eliglustat Drug–Drug Interactions.
- Published in:
- Clinical Pharmacology & Therapeutics, 2022, v. 112, n. 6, p. 1254, doi. 10.1002/cpt.2738
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- Publication type:
- Article
Neurochemical abnormalities in patients with type 1 Gaucher disease on standard of care therapy.
- Published in:
- Journal of Inherited Metabolic Disease, 2020, v. 43, n. 3, p. 564, doi. 10.1002/jimd.12182
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- Publication type:
- Article
Molecular Interactions between Gasotransmitters in Patients with Obstructive Sleep Apnea.
- Published in:
- International Journal of Translational Medicine, 2022, v. 2, n. 3, p. 408, doi. 10.3390/ijtm2030032
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- Publication type:
- Article
N-acetylcysteine Pharmacology and Applications in Rare Diseases—Repurposing an Old Antioxidant.
- Published in:
- Antioxidants, 2023, v. 12, n. 7, p. 1316, doi. 10.3390/antiox12071316
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- Publication type:
- Article
Orphan drug development: the increasing role of clinical pharmacology.
- Published in:
- Journal of Pharmacokinetics & Pharmacodynamics, 2019, v. 46, n. 5, p. 395, doi. 10.1007/s10928-019-09646-3
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- Publication type:
- Article
N -Acetylcysteine Reverses the Mitochondrial Dysfunction Induced by Very Long-Chain Fatty Acids in Murine Oligodendrocyte Model of Adrenoleukodystrophy.
- Published in:
- Biomedicines, 2021, v. 9, n. 12, p. 1826, doi. 10.3390/biomedicines9121826
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- Publication type:
- Article
N-acetylcysteine Provides Cytoprotection in Murine Oligodendrocytes through Heme Oxygenase-1 Activity.
- Published in:
- Biomedicines, 2020, v. 8, n. 8, p. 240, doi. 10.3390/biomedicines8080240
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- Publication type:
- Article
MicroRNAs as Biomarkers in Cancer.
- Published in:
- Diagnostics (2075-4418), 2013, v. 3, n. 1, p. 84, doi. 10.3390/diagnostics3010084
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- Publication type:
- Article
Population Pharmacokinetic Analysis of N‐Acetylcysteine in Pediatric Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplant.
- Published in:
- Journal of Clinical Pharmacology, 2021, v. 61, n. 12, p. 1638, doi. 10.1002/jcph.1943
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- Publication type:
- Article
Repeated‐Dose Oral N‐Acetylcysteine in Parkinson's Disease: Pharmacokinetics and Effect on Brain Glutathione and Oxidative Stress.
- Published in:
- Journal of Clinical Pharmacology, 2018, v. 58, n. 2, p. 158, doi. 10.1002/jcph.1008
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- Publication type:
- Article
Gaucher disease – more than just a rare lipid storage disease.
- Published in:
- Journal of Molecular Medicine, 2022, v. 100, n. 4, p. 499, doi. 10.1007/s00109-021-02174-z
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- Publication type:
- Article
Effects of yoga on oxidative stress, motor function, and non-motor symptoms in Parkinson's disease: a pilot randomized controlled trial.
- Published in:
- Pilot & Feasibility Studies, 2018, v. 4, n. 1, p. N.PAG, doi. 10.1186/s40814-018-0355-8
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- Publication type:
- Article