ReviewNutrients2021
Glycogen Storage Disease Type Ia: Current Management Options, Burden and Unmet Needs.
Review in Nutrients, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 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
47 citing papers in PubMed, 72 citations in OpenAlex.
- Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.Journal of inherited metabolic disease · 2026Trial
- Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).Journal of inherited metabolic disease · 2025Trial
- A Prospective Study on Continuous Glucose Monitoring in Glycogen Storage Disease Type Ia: Toward Glycemic Targets.The Journal of clinical endocrinology and metabolism · 2022Trial
- Towards mRNA therapeutics 2.0.Nature reviews. Drug discovery · 2026Review
- Hepatocyte-specific Cas9-mediated editing of G6pc and Slc37a4 elicits comparable biochemical and regulatory responses between glycogen storage disease (GSD) type Ia and Ib mice.Molecular metabolism · 2026Article
- Pregnancy in a patient with glycogen storage disease type Ia (von Gierke disease): case report and management strategy.AJOG global reports · 2026Article
- Two Classes of Protein Therapeutics: Why Dose-Response Architecture Defines the Boundary of mRNA Medicines.Pharmaceutics · 2026Review
- Health-Related Quality of Life in Pediatric Hepatic Glycogen Storage Disease: A Dual-Perspective Study.Nutrients · 2026Article
- A National Overview of Nutritional Care in Diet-Treated Inborn Errors of Metabolism in Brazil.International journal of environmental research and public health · 2026Article
- Successful surgical management of multiple hepatocellular adenomas in a young female patient with glycogen storage disease type Ia: a case report.Journal of medical case reports · 2026Article
- Continuous Glucose Monitoring-Driven Personalization of Cornstarch Therapy in Glycogen Storage Disease: A Retrospective Analysis.Yonsei medical journal · 2026Article
- Navigating Glycogen Storage Disease: The Spiritual and Cultural Journeys of Jordanian Mothers.Journal of religion and health · 2026Article
- Infantile extreme hypertriglyceridemia diagnosed as glycogen storage disease type Ia: A case report.Medicine · 2026Article
- Proteomics profiling of serum and liver in GSD Ia and Ib patients: insights into complication mechanisms and circulation biomarkers.Journal of translational medicine · 2026Article
- Applications and limitations of pluripotent stem cell-derived liver organoids.Chinese medical journal · 2026Review
- Development and Preliminary Psychometric Evaluation of the Glycogen Storage Disease Type Ia Functional Assessment Diary (GSD FAD).Patient preference and adherence · 2026Article
- Article
- Generation of a glycogen storage disease mouse model with fatty liver and its treatment by PPAR-α agonist bezafibrate.Frontiers in pharmacology · 2026Article
- Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy.Journal of health economics and outcomes research · 2026Article
- The induced-fit and catalytic mechanisms of human G6PC1.Cell discovery · 2025Article
Corrections and comments
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Authors and funding
15 authors at 11 institutions in 8 countries.
Funding
Abstract
Glycogen storage disease type Ia (GSDIa) is caused by defective glucose-6-phosphatase, a key enzyme in carbohydrate metabolism. Affected individuals cannot release glucose during fasting and accumulate excess glycogen and fat in the liver and kidney, putting them at risk of severe hypoglycaemia and secondary metabolic perturbations. Good glycaemic/metabolic control through strict dietary treatment and regular doses of uncooked cornstarch (UCCS) is essential for preventing hypoglycaemia and long-term complications. Dietary treatment has improved the prognosis for patients with GSDIa; however, the disease itself, its management and monitoring have significant physical, psychological and psychosocial burden on individuals and parents/caregivers. Hypoglycaemia risk persists if a single dose of UCCS is delayed/missed or in cases of gastrointestinal intolerance. UCCS therapy is imprecise, does not treat the cause of disease, may trigger secondary metabolic manifestations and may not prevent long-term complications. We review the importance of and challenges associated with achieving good glycaemic/metabolic control in individuals with GSDIa and how this should be balanced with age-specific psychosocial development towards independence, management of anxiety and preservation of quality of life (QoL). The unmet need for treatment strategies that address the cause of disease, restore glucose homeostasis, reduce the risk of hypoglycaemia/secondary metabolic perturbations and improve QoL is also discussed.
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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.