ArticleGenetics in medicine : official journal of the American College of Medical Genetics2019
Liver fibrosis during clinical ascertainment of glycogen storage disease type III: a need for improved and systematic monitoring.
Article in Genetics in medicine : official journal of the American College of Medical Genetics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 1 of them a synthesis that pooled it.
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Who cites it
35 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Dietary lipids in glycogen storage disease type III: A systematic literature study, case studies, and future recommendations.Journal of inherited metabolic disease · 2020Pooled it
- Effects of acute nutritional ketosis during exercise in adults with glycogen storage disease type IIIa are phenotype-specific: An investigator-initiated, randomized, crossover study.Journal of inherited metabolic disease · 2021Trial
- Highly potent MyoAAV4A vector reverses GSD III pathology in aged mice and enables long-term muscle disease correction in young adult mice.Molecular therapy. Advances · 2026Article
- Enhanced lysosomal glycogen breakdown is associated with liver tumorigenesis in glycogen storage disease type III.JHEP reports : innovation in hepatology · 2026Article
- Cell modeling and rescue of a novel noncoding genetic cause of glycogen storage disease IX.Genetics in medicine open · 2026Article
- Physical exercise intervention in glycogen storage disease IIIa: Feasibility and multisystem benefits.Experimental physiology · 2026Article
- Glycogen Storage Disease in Twins: When Two Lives Reflect One Silent Battle.Clinical case reports · 2026Article
- Evaluating and monitoring liver disease severity in glycogen storage disease type IX: Performance of novel and established clinical scores.Genetics in medicine open · 2026Article
- Weighted Gene Co-Expression Network Analysis and Alternative Splicing Analysis Reveal Key Genes Regulating Overfeeding-Induced Fatty Liver in Lion-Head Goose.International journal of molecular sciences · 2025Article
- High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa.Molecular therapy. Methods & clinical development · 2025Article
- Galectin-3: a novel biomarker of glycogen storage disease type III.Cell death discovery · 2025Article
- The relation between dietary polysaccharide intake and urinary excretion of tetraglucoside.Journal of inherited metabolic disease · 2025Article
- Progressive liver disease and dysregulated glycogen metabolism in murine GSD IX γ2 models human disease.Molecular genetics and metabolism · 2024Article
- [Genetic and clinical characteristics of 26 cases with glycogen storage disease type Ⅲ].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2024Article
- Hepatocyte-specific CCAAT/enhancer binding protein α restricts liver fibrosis progression.The Journal of clinical investigation · 2024Article
- A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.The Journal of clinical investigation · 2024Article
- Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024Review
- Glycogen storage diseases.Nature reviews. Disease primers · 2023Review
- Glycogen storage diseases: An update.World journal of gastroenterology · 2023Review
- Current understanding on pathogenesis and effective treatment of glycogen storage disease type Ib with empagliflozin: new insights coming from diabetes for its potential implications in other metabolic disorders.Frontiers in endocrinology · 2023Review
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeIn glycogen storage disease type III (GSD III), liver aminotransferases tend to normalize with age giving an impression that hepatic manifestations improve with age. However, despite dietary treatment, long-term liver complications emerge. We present a GSD III liver natural history study in children to better understand changes in hepatic parameters with age.
methodsWe reviewed clinical, biochemical, histological, and radiological data in pediatric patients with GSD III, and performed a literature review of GSD III hepatic findings.
resultsTwenty-six patients (median age 12.5 years, range 2-22) with GSD IIIa (n = 23) and IIIb (n = 3) were enrolled in the study. Six of seven pediatric patients showed severe fibrosis on liver biopsy (median [range] age: 1.25 [0.75-7] years). Markers of liver injury (aminotransferases), dysfunction (cholesterol, triglycerides), and glycogen storage (glucose tetrasaccharide, Glc
conclusionLiver fibrosis can occur at an early age, and may explain the decrease in aminotransferases and Glc
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