Waleed Alghamdi, Mahmoud Mosli, Saleh A. Alqahtani
{"title":"Gut microbiota in MAFLD: therapeutic and diagnostic implications","authors":"Waleed Alghamdi, Mahmoud Mosli, Saleh A. Alqahtani","doi":"10.1177/20420188241242937","DOIUrl":"https://doi.org/10.1177/20420188241242937","url":null,"abstract":"Metabolic dysfunction-associated fatty liver disease (MAFLD), formerly known as nonalcoholic fatty liver disease, is becoming a significant contributor to chronic liver disease globally, surpassing other etiologies, such as viral hepatitis. Prevention and early treatment strategies to curb its growing prevalence are urgently required. Recent evidence suggests that targeting the gut microbiota may help treat and alleviate disease progression in patients with MAFLD. This review aims to explore the complex relationship between MAFLD and the gut microbiota in relation to disease pathogenesis. Additionally, it delves into the therapeutic strategies targeting the gut microbiota, such as diet, exercise, antibiotics, probiotics, synbiotics, glucagon-like peptide-1 receptor agonists, and fecal microbiota transplantation, and discusses novel biomarkers, such as microbiota-derived testing and liquid biopsy, for their diagnostic and staging potential. Overall, the review emphasizes the urgent need for preventive and therapeutic strategies to address the devastating consequences of MAFLD at both individual and societal levels and recognizes that further exploration of the gut microbiota may open avenues for managing MAFLD effectively in the future.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140584930","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Age-related markers and predictors of diabetic kidney disease progression in type 2 diabetes patients: a retrospective cohort study","authors":"Larissa Fabre, Érika Bevilaqua Rangel","doi":"10.1177/20420188241242947","DOIUrl":"https://doi.org/10.1177/20420188241242947","url":null,"abstract":"Background:Diabetic kidney disease (DKD) is characterized by reduced estimated glomerular filtration rate (eGFR) and albuminuria, which play a pivotal role in both diagnosing and determining the disease’s progression. This study aimed to assess the trajectory of these markers concerning age in individuals with DKD and identify predictive factors for the decline in eGFR decline, variation in albuminuria, mortality, and progression to renal replacement therapy (RRT).Design:This retrospective cohort encompassed patients with type 2 diabetes (T2D), divided into two age categories: <75 and ⩾75 years old.Methods:Over a 3-year span, the study evaluated eGFR (CKD-EPI) and 24-h albuminuria. Univariate and multivariate analyses were employed to pinpoint factors associated with deteriorating renal function and mortality. Significance was set at p < 0.05, and Kaplan–Meier survival curves were constructed to illustrate renal and overall survival.Results:The analysis comprised 304 patients. Comparable eGFR declines were evident in both age groups during the transition from the first to the second year and from the second to the third year. Nonetheless, a more pronounced rise in albuminuria was evident in the ⩾75 years group during the first to the second year. Multivariate analysis unveiled that systolic blood pressure (SBP) measurements in the first year positively forecasted eGFR decline. Age was associated with heightened albuminuria and mortality, while hospitalizations linked to cardiovascular causes robustly predicted mortality. Hospitalizations due to sepsis and cardiovascular reasons, coupled with first-year SBP measurements, served as predictive indicators for progression to RRT.Conclusion:Both age groups experienced similar declines in eGFR, though the ⩾75 years group displayed a more significant increase in albuminuria during the first to the second year. Age, hospitalizations, and higher blood pressure levels were correlated with exacerbated renal function deterioration and/or elevated mortality in DKD. Timely intervention and tailored management strategies stand as critical components for enhancing outcomes among DKD patients.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140585006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sindhoora Kotha, Sven Plein, John P. Greenwood, Eylem Levelt
{"title":"Role of epicardial adipose tissue in diabetic cardiomyopathy through the lens of cardiovascular magnetic resonance imaging – a narrative review","authors":"Sindhoora Kotha, Sven Plein, John P. Greenwood, Eylem Levelt","doi":"10.1177/20420188241229540","DOIUrl":"https://doi.org/10.1177/20420188241229540","url":null,"abstract":"Accumulating evidence suggests that ectopic/visceral adiposity may play a key role in the pathogenesis of nonischaemic cardiovascular diseases associated with type 2 diabetes. Epicardial adipose tissue (EAT) is a complex visceral fat depot, covering 80% of the cardiac surface with anatomical and functional contiguity to the myocardium and coronary arteries. EAT interacts with the biology of the underlying myocardium by secreting a wide range of adipokines. Magnetic resonance imaging (MRI) is the reference modality for structural and functional imaging of the heart. The technique is now also emerging as the reference imaging modality for EAT quantification. With this narrative review, we (a) surveyed contemporary clinical studies that utilized cardiovascular MRI to characterize EAT (studies published 2010–2023); (b) listed the clinical trials monitoring the response to treatment in EAT size as well as myocardial functional and structural parameters and (c) discussed the potential pathophysiological role of EAT in the development of diabetic cardiomyopathy. We concluded that increased EAT quantity and its inflammatory phenotype correlate with early signs of left ventricle dysfunction and may have a role in the pathogenesis of cardiac disease in diabetes with and without coronary artery disease.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140106320","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Riemer A. Been, Annel Lameijer, Reinold O. B. Gans, André P. van Beek, Andrew P. Kingsnorth, Pratik Choudhary, Peter R. van Dijk
{"title":"The impact of socioeconomic factors, social determinants, and ethnicity on the utilization of glucose sensor technology among persons with diabetes mellitus: a narrative review","authors":"Riemer A. Been, Annel Lameijer, Reinold O. B. Gans, André P. van Beek, Andrew P. Kingsnorth, Pratik Choudhary, Peter R. van Dijk","doi":"10.1177/20420188241236289","DOIUrl":"https://doi.org/10.1177/20420188241236289","url":null,"abstract":"Continuous glucose monitoring (CGM) usage has been shown to improve disease outcomes in people living with diabetes by facilitating better glycemic management. However, previous research has suggested that access to these devices can be influenced by nonmedical factors such as socioeconomic status and ethnicity. It is critical that equitable access to CGM devices is ensured as people from those groups experience poorer diabetes-related health outcomes. In this narrative review, we provide an overview of the various healthcare systems worldwide and how socioeconomic status, social context, and ethnicity shape device usage and the associated health outcomes. In general, we found that having a lower socioeconomic status and belonging to an ethnic minority group negatively impact CGM usage. While financial means proved to be an important mediator in this process, it was not the sole driver as disparities persisted even after adjustment for factors such as income and insurance status. Recommendations to increase CGM usage for people of a lower socioeconomic status and ethnic minorities include increasing the availability of financial, administrative, and educational support, for both patients and healthcare providers. However, recommendations will vary due to local country-specific circumstances, such as reimbursement criteria and healthcare ecosystems.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140106170","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Epidemiology of gout - Global burden of disease research from 1990 to 2019 and future trend predictions.","authors":"Tingfen Han, Wenli Chen, Xiasang Qiu, Weijie Wang","doi":"10.1177/20420188241227295","DOIUrl":"10.1177/20420188241227295","url":null,"abstract":"<p><strong>Background: </strong>Understanding the global burden of gout in the past and future can provide important references for optimizing prevention and control strategies in healthcare systems.</p><p><strong>Objectives: </strong>This study aimed to report variations in the global disease burden and risk factors of gout in 204 countries and territories from 1990 to 2019.</p><p><strong>Design: </strong>We conducted a retrospective analysis of gout based on the latest Global Burden of Disease (GBD) 2019 database.</p><p><strong>Methods: </strong>We collected data on the prevalence, incidence, and disability-adjusted life years (DALYs) of gout from 1990 to 2019. The data were then stratified by age, sex, and economic development level. Decomposition analysis, frontier analysis, and prediction models were used to analyze the changes and influencing factors influencing each indicator.</p><p><strong>Results: </strong>Globally, there were 53,871,846.4 [95% uncertainty interval (UI): 43,383,204.6-66,342,327.3] prevalent cases, 92,228,86.8 (95% UI: 7419,132.1-11,521,165) incident cases, and 1673,973.4 (95% UI: 1,068,061.1-2,393,469.2) cases of DALYs of gout in 2019, more than double those in 1990. Moreover, the pace of increase in the age-standardized prevalence rate (ASPR), age-standardized incidence rate (ASIR), and age-standardized DALY rate (ASDR) accelerated during 1990-2019, with estimated annual percentage changes (EAPC) of 0.94 [95% confidence interval (CI): 0.85-1.03], 0.77 (95% CI: 0.69-0.84), and 0.93 (95% CI: 0.84-1.02), respectively, especially among men. The disease burden of gout has increased in all the other 20 GBD regions in the past 30 years, except Western Sub-Saharan Africa. The highest risk of high body mass index (BMI) and kidney dysfunction was in high-income countries such as North America and East Asia. The global prevalence rate, incidence rate, and DALYs rate of gout in 2030 will reach 599.86, 102.96 per 100,000 population, and 20.26 per 100,000 population, respectively, roughly the same as in 2019.</p><p><strong>Conclusion: </strong>With the development of society, the disease burden of gout will become increasingly severe. It is very important to study the accurate epidemiological data on gout for clinical diagnosis and treatment and health policy.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10910883/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140029068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ritah Kiconco, Sylvia Achieng Lumumba, Charles Nkubi Bagenda, Raymond Atwine, Joseph Ndarubweine, Simon Peter Rugera
{"title":"Insulin therapy among diabetic patients in rural communities of Sub-Saharan Africa: a perspective review.","authors":"Ritah Kiconco, Sylvia Achieng Lumumba, Charles Nkubi Bagenda, Raymond Atwine, Joseph Ndarubweine, Simon Peter Rugera","doi":"10.1177/20420188241232280","DOIUrl":"10.1177/20420188241232280","url":null,"abstract":"<p><p>In this perspective review, we describe a brief background on the status quo of diabetes mellitus-related therapies and glycemic control among patients in rural communities in sub-Saharan Africa. The article discusses insulin therapy as well as the difficulties in obtaining insulin and oral hypoglycemic medications for diabetic patients living in sub-Saharan Africa. We wrap up our discussion with suggestions on solutions and opportunities for future research to tackle this health challenge in these impoverished communities. We conducted a literature search from PubMed and Google Scholar up until August 2023. Key words were used to generate search terms used to retrieve the required information. All types of literature with pertinent information on the current topic were included in the study. Diabetes mellitus is on the rise in sub-Saharan Africa. Several studies have reported poor glycemic control, low screening rates for diabetes mellitus, cigarette smoking, high alcohol consumption, prescription of antidiabetic therapy, and associated costs as contributors to the uptake of antidiabetic treatment. Although there is paucity of data on the extent of insulin therapy uptake and its possible modifiable contributors among the diabetic patients in the region, the anticipated increase in the number of people with diabetes on the continent makes it critical for global leaders to address the research gaps in insulin therapy among rural communities of sub-Saharan Africa, thus reducing the burden of diabetes in these populations.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2024-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10878220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139913473","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Consolato M Sergi, Mohit Kehar, Carolina Jimenez-Rivera
{"title":"Liver Biopsy Handling of Metabolic-Associated Fatty Liver Disease (MAFLD): the Children's Hospital of Eastern Ontario grossing protocol.","authors":"Consolato M Sergi, Mohit Kehar, Carolina Jimenez-Rivera","doi":"10.1177/20420188241227766","DOIUrl":"10.1177/20420188241227766","url":null,"abstract":"<p><p>Metabolic-(non-alcoholic) associated fatty liver disease (MAFLD/NAFLD) has increasingly become a worldwide epidemic. It has been suggested that renaming NAFLD to MAFLD is critical in identifying patients with advanced fibrosis and poor cardiovascular outcomes. There are concerns that the progression to non-alcoholic steatohepatitis (NASH) may become a constant drive in the future healthcare of children and adolescents. There is a necessity to tackle the emerging risk factors for NASH-associated hepatocellular carcinoma (HCC). In this narrative review, we present the current protocol of liver biopsy separated between pre-analytical, analytical, and post-analytical handling. Genetic association investigations have identified single nucleotide polymorphisms implicated in the progression of MAFLD-HCC, many of which seem to belong to the lipid metabolism pathways. PNPLA3 rs738409 variant, TM6SF2 rs58542926 variant, MBOAT7 rs641738 variant, and GCKR variants seem to be significantly associated with NAFLD disease susceptibility. In disclosing the current comprehensive protocol performed at the Children's Hospital of Eastern Ontario, Ottawa, ON, Canada, we support the most recent Kulkarni-Sarin's pledge to rename NAFLD to MAFLD. Grossing of the liver biopsy is key to identifying histologic, immunophenotypical, and ultrastructure data and properly preserving tissue for molecular genomics data.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10846056/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139698384","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Anti-inflammatory role of glucagon-like peptide 1 receptor agonists and its clinical implications.","authors":"Saleh Hadi Alharbi","doi":"10.1177/20420188231222367","DOIUrl":"10.1177/20420188231222367","url":null,"abstract":"<p><p>Glucagon-like peptide 1 receptor agonists (GLP-1RAs) have emerged as promising therapeutic agents with potent anti-inflammatory properties and diverse clinical implications. This in-depth review article explores the mechanisms behind the anti-inflammatory actions of GLP-1RAs and assesses their prospective applicability in a wide range of disease scenarios. The current review establishes the significance of comprehending the anti-inflammatory role of GLP-1RAs and identifies pertinent research gaps. A concise overview of inflammation and its clinical consequences underscores the critical need for effective anti-inflammatory interventions. Subsequently, the article elucidates the intricate mechanisms through which GLP-1RAs modulate immune cell signaling and regulate the nuclear factor-kappa B (NF-κB) pathway. Detailed discussions encompass their impact on inflammatory responses, cytokine production, and attenuation of oxidative stress. The exposition is substantiated by a collection of pertinent examples and an extensive array of references from both preclinical and clinical investigations. The historical trajectory of GLP-1RA drugs, including exenatide, lixisenatide, liraglutide, and semaglutide, is traced to delineate their development as therapeutic agents. Moreover, the review emphasizes the therapeutic potential of GLP-1RAs in specific disease contexts like type 2 diabetes, a neurodegenerative disorder, and inflammatory bowel disease (IBD), shedding light on their anti-inflammatory effects through rigorous examination of preclinical and clinical studies. The article also provides an outlook on future perspectives for GLP-1RAs, encompassing the domains of diabetes, neurodegenerative diseases, and IBD. In conclusion, GLP-1RAs exhibit substantial anti-inflammatory effects, rendering them promising therapeutic agents with broad clinical implications. They are very useful in a wide variety of diseases because they regulate immunological responses, block NF-κB activation, and decrease production of pro-inflammatory cytokines. Ongoing research endeavors aim to optimize their therapeutic use, delineate patient-specific treatment paradigms, and explore novel therapeutic applications. GLP-1RAs represent a significant breakthrough in anti-inflammatory therapy, offering novel treatment options, and improved patient outcomes.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2024-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10823863/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139576261","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Therapeutic potential of adiponectin in prediabetes: strategies, challenges, and future directions.","authors":"Mona Mohamed Ibrahim Abdalla","doi":"10.1177/20420188231222371","DOIUrl":"10.1177/20420188231222371","url":null,"abstract":"<p><p>Adiponectin, an adipose-derived hormone, plays a pivotal role in glucose regulation and lipid metabolism, with a decrease in circulating adiponectin levels being linked to insulin resistance and prediabetes. This review examines the therapeutic potential of adiponectin in managing prediabetes, elucidating on multiple aspects including its role in glucose and lipid metabolism, influence on insulin sensitivity, and anti-inflammatory properties. Moreover, the paper highlights the latest strategies to augment adiponectin levels, such as gene therapy, pharmacological interventions, dietary modifications, and lifestyle changes. It also addresses the challenges encountered in translating preclinical findings into clinical practice, primarily related to drug delivery, safety, and efficacy. Lastly, the review proposes future directions, underlining the need for large-scale human trials, novel adiponectin analogs, and personalized treatment strategies to harness adiponectin's full therapeutic potential in preventing the transition from prediabetes to diabetes.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2024-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10798122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139512126","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ayla Evins, Jill Mayhew, Tricia Cimms, Julie Whyte, Kathy Vong, Elizabeth Hribal, Christopher J Evans, Andrew Grimm
{"title":"Glycogen storage disease type III: a mixed-methods study to assess the burden of disease.","authors":"Ayla Evins, Jill Mayhew, Tricia Cimms, Julie Whyte, Kathy Vong, Elizabeth Hribal, Christopher J Evans, Andrew Grimm","doi":"10.1177/20420188231224233","DOIUrl":"10.1177/20420188231224233","url":null,"abstract":"<p><strong>Background: </strong>Glycogen storage disease type III (GSD III) is a rare inherited disorder that results from a glycogen debranching enzyme deficiency.</p><p><strong>Objectives: </strong>The purpose of this research was to collect data on the signs, symptoms, and impacts of GSD III from the perspective of adult patients and caregivers of individuals with GSD III.</p><p><strong>Design: </strong>Online survey and qualitative interviews.</p><p><strong>Methods: </strong>Following institutional review board approval, adult patients and caregivers of children with GSD III were recruited through advocacy networks and clinical sites. If eligible, participants were consented, screened, and sent a survey and/or participated in a 60-min interview. The survey and interview included questions about family history, diagnosis, signs and symptoms, impacts, and management of GSD III. Conceptual models were developed following the analysis of results.</p><p><strong>Results: </strong>In all, 29 adults and 46 caregivers completed the online survey and/or the interviews with 73 survey and 19 interview respondents. Adults and caregivers reported digestive, musculoskeletal, growth and physical appearance, and cardiac signs and symptoms. Liver conditions were reported by most respondents (83%). Adults and caregivers frequently reported impacts such as difficulty keeping up with peers (77%) and difficulty exercising/difficulty with physical activity (53%). Hypoglycemia was frequently reported in both adults and children, with more than half reporting hospitalizations due to hypoglycemia. Caregivers focused on hypoglycemia when reporting signs/symptoms that most interfere with their child's life and prevention of hypoglycemia as a desired outcome for an effective therapy. Adults most often reported muscle weakness as a top interfering symptom and the most important goal of a potential therapy. Impacts were also reported in activities of daily living, cognitive, emotional, work/school, and sleep domains.</p><p><strong>Conclusion: </strong>Individuals with GSD III experience a broad spectrum of symptoms and disease impacts. There is an unmet need for therapies that improve metabolic control, reduce the burden of dietary management, reduce fatigue and liver problems, and improve muscle strength and function.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10775738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139404500","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}