Degenerative neurological and neuromuscular disease最新文献

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Advances in Blood Biomarkers for Alzheimer's Disease: Ultra-Sensitive Detection Technologies and Impact on Clinical Diagnosis. 阿尔茨海默病血液生物标志物研究进展:超灵敏检测技术及其对临床诊断的影响。
IF 2.1
Degenerative neurological and neuromuscular disease Pub Date : 2024-07-30 eCollection Date: 2024-01-01 DOI: 10.2147/DNND.S471174
Yi Zhang, Kefan Bi, Linfu Zhou, Jie Wang, Lingtong Huang, Yan Sun, Guoping Peng, Wei Wu
{"title":"Advances in Blood Biomarkers for Alzheimer's Disease: Ultra-Sensitive Detection Technologies and Impact on Clinical Diagnosis.","authors":"Yi Zhang, Kefan Bi, Linfu Zhou, Jie Wang, Lingtong Huang, Yan Sun, Guoping Peng, Wei Wu","doi":"10.2147/DNND.S471174","DOIUrl":"10.2147/DNND.S471174","url":null,"abstract":"<p><p>Alzheimer's disease has escalated into a critical public health concern, marked by its neurodegenerative nature that progressively diminishes cognitive abilities. Recognized as a continuously advancing and presently incurable condition, AD underscores the necessity for early-stage diagnosis and interventions aimed at delaying the decline in mental function. Despite the proven efficacy of cerebrospinal fluid and positron emission tomography in diagnosing AD, their broader utility is constrained by significant costs and the invasive nature of these procedures. Consequently, the innovation of blood biomarkers such as Amyloid-beta, phosphorylated-tau, total-tau et al, distinguished by their high sensitivity, minimal invasiveness, accessibility, and cost-efficiency, emerges as a promising avenue for AD diagnosis. The advent of ultra-sensitive detection methodologies, including single-molecule enzyme-linked immunosorbent assay and immunoprecipitation-mass spectrometry, has revolutionized the detection of AD plasma biomarkers, supplanting previous low-sensitivity techniques. This rapid advancement in detection technology facilitates the more accurate quantification of pathological brain proteins and AD-associated biomarkers in the bloodstream. This manuscript meticulously reviews the landscape of current research on immunological markers for AD, anchored in the National Institute on Aging-Alzheimer's Association AT(N) research framework. It highlights a selection of forefront ultra-sensitive detection technologies now integral to assessing AD blood immunological markers. Additionally, this review examines the crucial pre-analytical processing steps for AD blood samples that significantly impact research outcomes and addresses the practical challenges faced during clinical testing. These discussions are crucial for enhancing our comprehension and refining the diagnostic precision of AD using blood-based biomarkers. The review aims to shed light on potential avenues for innovation and improvement in the techniques employed for detecting and investigating AD, thereby contributing to the broader field of neurodegenerative disease research.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11297492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141891289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Childhood Cerebral Adrenoleukodystrophy: Case Report and Literature Review Advocating for Newborn Screening. 儿童脑性肾上腺白质营养不良症:病例报告和文献综述,倡导新生儿筛查。
IF 2.1
Degenerative neurological and neuromuscular disease Pub Date : 2024-06-17 eCollection Date: 2024-01-01 DOI: 10.2147/DNND.S442985
Hamrish Kumar Rajakumar, Varsha Coimbatore Sathyabal, Revathi Nachiappan, Sivakumar Krishnaswamy Vijayaramanujam
{"title":"Childhood Cerebral Adrenoleukodystrophy: Case Report and Literature Review Advocating for Newborn Screening.","authors":"Hamrish Kumar Rajakumar, Varsha Coimbatore Sathyabal, Revathi Nachiappan, Sivakumar Krishnaswamy Vijayaramanujam","doi":"10.2147/DNND.S442985","DOIUrl":"10.2147/DNND.S442985","url":null,"abstract":"<p><strong>Background: </strong>X-linked adrenoleukodystrophy (ALD) is a rare genetic disorder caused by a pathogenic variant of the ABCD1 gene, leading to impaired peroxisomal function and the accumulation of very long-chain fatty acids (VLCFAs). ALD presents a wide range of neurological and adrenal symptoms, ranging from childhood cerebral adrenoleukodystrophy to adrenomyeloneuropathy and adrenal insufficiency. Newborn screening (NBS) for ALD is available in some regions but remains lacking in others, such as India.</p><p><strong>Case presentation: </strong>We present a case of a 10-year-old boy with ALD who presented with seizures, progressive weakness, visual impairment, and adrenal insufficiency. Despite symptomatic management and dietary adjustments, the disease progressed rapidly, leading to respiratory failure and eventual demise. The diagnosis was confirmed through molecular analysis and elevated VLCFA levels. Neuroimaging revealed characteristic white matter changes consistent with ALD.</p><p><strong>Conclusion: </strong>ALD is a devastating disease with no cure, emphasizing the importance of early detection through newborn screening and genetic testing. Management strategies include adrenal hormone therapy, gene therapy, and allogenic stem cell transplantation, as well as investigational treatments such as VLCFA normalization. Our case advocates the need for worldwide NBS and pediatric neurologic follow-up to enable early intervention and improve patient outcomes. Additionally, the association between ALD, recurrent febrile seizures, and unexplained developmental delay warrants further investigation to better understand disease progression and potential therapeutic targets.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2024-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11192191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141443969","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Review of Pharmacotherapeutic Targets in Alzheimer's Disease and Its Management Using Traditional Medicinal Plants. 阿尔茨海默病的药物治疗靶点及其利用传统药用植物进行管理的综述。
Degenerative neurological and neuromuscular disease Pub Date : 2024-05-19 eCollection Date: 2024-01-01 DOI: 10.2147/DNND.S452009
Prabhash Nath Tripathi, Ankit Lodhi, Sachchida Nand Rai, Nilay Kumar Nandi, Shweta Dumoga, Pooja Yadav, Amit Kumar Tiwari, Santosh Kumar Singh, Abdel-Nasser A El-Shorbagi, Sachin Chaudhary
{"title":"Review of Pharmacotherapeutic Targets in Alzheimer's Disease and Its Management Using Traditional Medicinal Plants.","authors":"Prabhash Nath Tripathi, Ankit Lodhi, Sachchida Nand Rai, Nilay Kumar Nandi, Shweta Dumoga, Pooja Yadav, Amit Kumar Tiwari, Santosh Kumar Singh, Abdel-Nasser A El-Shorbagi, Sachin Chaudhary","doi":"10.2147/DNND.S452009","DOIUrl":"10.2147/DNND.S452009","url":null,"abstract":"<p><p>Alzheimer's disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline, memory loss, and impaired daily functioning. While there is currently no cure for AD, several pharmacotherapeutic targets and management strategies have been explored. Additionally, traditional medicinal plants have gained attention for their potential role in AD management. Pharmacotherapeutic targets in AD include amyloid-beta (Aβ) aggregation, tau protein hyperphosphorylation, neuroinflammation, oxidative stress, and cholinergic dysfunction. Traditional medicinal plants, such as <i>Ginkgo biloba, Huperzia serrata, Curcuma longa</i> (turmeric), and Panax ginseng, have demonstrated the ability to modulate these targets through their bioactive compounds. <i>Ginkgo biloba</i>, for instance, contains flavonoids and terpenoids that exhibit neuroprotective effects by reducing Aβ deposition and enhancing cerebral blood flow. <i>Huperzia serrata</i>, a natural source of huperzine A, has acetylcholinesterase-inhibiting properties, thus improving cholinergic function. <i>Curcuma longa</i>, enriched with curcumin, exhibits anti-inflammatory and antioxidant effects, potentially mitigating neuroinflammation and oxidative stress. Panax ginseng's ginsenosides have shown neuroprotective and anti-amyloidogenic properties. The investigation of traditional medicinal plants as a complementary approach to AD management offers several advantages, including a lower risk of adverse effects and potential multi-target interactions. Furthermore, the cultural knowledge and utilization of these plants provide a rich source of information for the development of new therapies. However, further research is necessary to elucidate the precise mechanisms of action, standardize preparations, and assess the safety and efficacy of these natural remedies. Integrating traditional medicinal-plant-based therapies with modern pharmacotherapies may hold the key to a more comprehensive and effective approach to AD treatment. This review aims to explore the pharmacotherapeutic targets in AD and assess the potential of traditional medicinal plants in its management.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11114142/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141089573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Comprehensive Review and Androgen Deprivation Therapy and Its Impact on Alzheimer’s Disease Risk in Older Men with Prostate Cancer 全面回顾雄激素剥夺疗法及其对老年男性前列腺癌患者阿尔茨海默病风险的影响
Degenerative neurological and neuromuscular disease Pub Date : 2024-05-01 DOI: 10.2147/dnnd.s445130
Manisha Singh, Vinayak Agarwal, Pranav Pancham, Divya Jindal, S. Agarwal, Sachchida Rai, Santosh Singh, Vivek Gupta
{"title":"A Comprehensive Review and Androgen Deprivation Therapy and Its Impact on Alzheimer’s Disease Risk in Older Men with Prostate Cancer","authors":"Manisha Singh, Vinayak Agarwal, Pranav Pancham, Divya Jindal, S. Agarwal, Sachchida Rai, Santosh Singh, Vivek Gupta","doi":"10.2147/dnnd.s445130","DOIUrl":"https://doi.org/10.2147/dnnd.s445130","url":null,"abstract":"","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141049078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Serum Exosomal miRNA-125b and miRNA-451a are Potential Diagnostic Biomarker for Alzheimer’s Diseases 血清外泌体 miRNA-125b 和 miRNA-451a 是阿尔茨海默病的潜在诊断生物标记物
Degenerative neurological and neuromuscular disease Pub Date : 2024-04-01 DOI: 10.2147/DNND.S444567
Xian Duan, Qing Zheng, Lihui Liang, Lin Zhou
{"title":"Serum Exosomal miRNA-125b and miRNA-451a are Potential Diagnostic Biomarker for Alzheimer’s Diseases","authors":"Xian Duan, Qing Zheng, Lihui Liang, Lin Zhou","doi":"10.2147/DNND.S444567","DOIUrl":"https://doi.org/10.2147/DNND.S444567","url":null,"abstract":"Aim To explore the diagnostic value of serum-derived exosomal miRNAs and predict the roles of their target genes in Alzheimer’s disease (AD) based on the expression of miRNAs in AD patients. Methods We determined the relative concentration of exosomal miRNAs by High-throughput Second-generation Sequencing and real-time quantitative real-time PCR. Results 71 AD patients and 71 ND subjects were collected. The study demonstrated that hsa-miR-125b-1-3p, hsa-miR-193a-5p, hsa-miR-378a-3p, hsa-miR-378i and hsa-miR-451a are differentially expressed in the serum-derived exosomes of AD patients compared with healthy subjects. According to ROC analysis, hsa-miR-125b-1-3p has an AUC of 0.765 in the AD group compared to the healthy group with a sensitivity and specificity of 82.1–67.7%, respectively. Enrichment analysis of its target genes showed that they were related to neuroactive ligand-receptor interactions, the PI3K-Akt signaling pathway, the Hippo signaling pathway and nervous system-related pathways. And, hsa-miR-451a had an AUC of 0.728 that differentiated the AD group from the healthy group with a sensitivity and specificity of 67.9% and 72.6%, respectively. Enrichment analysis of its target genes showed a relationship with cytokine-cytokine receptor interactions and the PI3K-Akt signaling pathway. Conclusion The dysregulation of serum exosomal microRNAs in patients with AD may promote the diagnosis of AD. The target genes of miRNAs may be involved in the occurrence and development of AD through various pathways.","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140775692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Transient Neonatal Myasthenia Gravis Born to a Mother with Asymptomatic MG: A Case Report. 无症状肌无力母亲所生的一过性新生儿肌无力:病例报告。
Degenerative neurological and neuromuscular disease Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.2147/DNND.S451611
Jinrong Yang, Liping Pan, Yaping Liu, Yanrong Wang
{"title":"Transient Neonatal Myasthenia Gravis Born to a Mother with Asymptomatic MG: A Case Report.","authors":"Jinrong Yang, Liping Pan, Yaping Liu, Yanrong Wang","doi":"10.2147/DNND.S451611","DOIUrl":"10.2147/DNND.S451611","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an autoimmune disease which can impact pregnancy. We describe a transient neonatal myasthenia gravis (TNMG) born to an asymptomatic mother aged 26. The newborn presented cyanosis and generalized muscular weakness quickly after birth. Nasal continuous positive airway pressure (nCPAP) ventilation was performed immediately. On day 3, detailed family history showed that the neonate's 50-year-old maternal grandmother was diagnosed as ocular MG at the age of 40. Ryanodine receptor calcium release channel antibody (RyR-Ab) and acetylcholine receptor antibody (AChR-Ab) tested on day 5 were positive. However, neostigmine tests were negative for the neonate. Intravenous immunoglobulin (IVIG) and oral pyridostigmine were administered. The infant was weaned from the ventilator on day 7. On day 10, the neonate's asymptomatic mother was confirmed to have positive AChR-Ab either. The neonate regained the capability of bottle feeding on day 17 and discharged on day 26. Asymptomatic pregnant women with MG family history can also deliver infants who develop TNMG. Testing AChR antibodies in pregnant women with a family history of MG should be necessary as TNMG was a life-threatening disease. With timely diagnosis and accurate treatment, TNMG can be effectively relieved.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10913793/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140041135","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current Perspectives: Obesity and Neurodegeneration - Links and Risks. 当前视角:肥胖与神经变性--联系与风险。
Degenerative neurological and neuromuscular disease Pub Date : 2023-12-31 eCollection Date: 2023-01-01 DOI: 10.2147/DNND.S388579
Paul J Kueck, Jill K Morris, John A Stanford
{"title":"Current Perspectives: Obesity and Neurodegeneration - Links and Risks.","authors":"Paul J Kueck, Jill K Morris, John A Stanford","doi":"10.2147/DNND.S388579","DOIUrl":"10.2147/DNND.S388579","url":null,"abstract":"<p><p>Obesity is increasing in prevalence across all age groups. Long-term obesity can lead to the development of metabolic and cardiovascular diseases through its effects on adipose, skeletal muscle, and liver tissue. Pathological mechanisms associated with obesity include immune response and inflammation as well as oxidative stress and consequent endothelial and mitochondrial dysfunction. Recent evidence links obesity to diminished brain health and neurodegenerative diseases such as Alzheimer's disease (AD) and Parkinson's disease (PD). Both AD and PD are associated with insulin resistance, an underlying syndrome of obesity. Despite these links, causative mechanism(s) resulting in neurodegenerative disease remain unclear. This review discusses relationships between obesity, AD, and PD, including clinical and preclinical findings. The review then briefly explores nonpharmacological directions for intervention.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10774290/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139405699","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Development and Validation of a Risk Prediction Model to Estimate the Risk of Stroke Among Hypertensive Patients in University of Gondar Comprehensive Specialized Hospital, Gondar, 2012 to 2022. 开发和验证风险预测模型,以估算贡德尔大学综合专科医院高血压患者的中风风险,2012-2022 年。
Degenerative neurological and neuromuscular disease Pub Date : 2023-12-14 eCollection Date: 2023-01-01 DOI: 10.2147/DNND.S435806
Yazachew Moges Chekol, Mehari Woldemariam Merid, Getayeneh Antehunegn Tesema, Tigabu Kidie Tesfie, Tsion Mulat Tebeje, Negalegn Byadgie Gelaw, Nebiyu Bekele Gebi, Wullo Sisay Seretew
{"title":"Development and Validation of a Risk Prediction Model to Estimate the Risk of Stroke Among Hypertensive Patients in University of Gondar Comprehensive Specialized Hospital, Gondar, 2012 to 2022.","authors":"Yazachew Moges Chekol, Mehari Woldemariam Merid, Getayeneh Antehunegn Tesema, Tigabu Kidie Tesfie, Tsion Mulat Tebeje, Negalegn Byadgie Gelaw, Nebiyu Bekele Gebi, Wullo Sisay Seretew","doi":"10.2147/DNND.S435806","DOIUrl":"10.2147/DNND.S435806","url":null,"abstract":"<p><strong>Background: </strong>A risk prediction model to predict the risk of stroke has been developed for hypertensive patients. However, the discriminating power is poor, and the predictors are not easily accessible in low-income countries. Therefore, developing a validated risk prediction model to estimate the risk of stroke could help physicians to choose optimal treatment and precisely estimate the risk of stroke.</p><p><strong>Objective: </strong>This study aims to develop and validate a risk prediction model to estimate the risk of stroke among hypertensive patients at the University of Gondar Comprehensive Specialized Hospital.</p><p><strong>Methods: </strong>A retrospective follow-up study was conducted among 743 hypertensive patients between September 01/2012 and January 31/2022. The participants were selected using a simple random sampling technique. Model performance was evaluated using discrimination, calibration, and Brier scores. Internal validity and clinical utility were evaluated using bootstrapping and a decision curve analysis.</p><p><strong>Results: </strong>Incidence of stroke was 31.4 per 1000 person-years (95% CI: 26.0, 37.7). Combinations of six predictors were selected for model development (sex, residence, baseline diastolic blood pressure, comorbidity, diabetes, and uncontrolled hypertension). In multivariable logistic regression, the discriminatory power of the model was 0.973 (95% CI: 0.959, 0.987). Calibration plot illustrated an overlap between the probabilities of the predicted and actual observed risks after 10,000 times bootstrap re-sampling, with a sensitivity of 92.79%, specificity 93.51%, and accuracy of 93.41%. The decision curve analysis demonstrated that the net benefit of the model was better than other intervention strategies, starting from the initial point.</p><p><strong>Conclusion: </strong>An internally validated, accurate prediction model was developed and visualized in a nomogram. The model is then changed to an offline mobile web-based application to facilitate clinical applicability. The authors recommend that other researchers eternally validate the model.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10728309/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138806821","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Practical Guidance on the Use of Cladribine Tablets in the Management or Relapsing Multiple Sclerosis: Expert Opinion from Qatar. 关于使用克拉利宾片治疗复发性多发性硬化症的实用指南:来自卡塔尔的专家意见。
Degenerative neurological and neuromuscular disease Pub Date : 2023-12-13 eCollection Date: 2023-01-01 DOI: 10.2147/DNND.S433459
Dirk Deleu, Beatriz Garcia Canibano, Osama Elalamy, Mohamed Sayed Abdelmoneim, Amir Boshra
{"title":"Practical Guidance on the Use of Cladribine Tablets in the Management or Relapsing Multiple Sclerosis: Expert Opinion from Qatar.","authors":"Dirk Deleu, Beatriz Garcia Canibano, Osama Elalamy, Mohamed Sayed Abdelmoneim, Amir Boshra","doi":"10.2147/DNND.S433459","DOIUrl":"https://doi.org/10.2147/DNND.S433459","url":null,"abstract":"<p><p>The increasing availability of high-efficacy disease-modifying therapies (DMT) for the management of relapsing multiple sclerosis (RMS) has increased the potential for individualised patient management but has added complexity to the design of treatment regimens. The long-term application of immune reconstitution therapy (IRT) is supported by an increasing database of real world studies that have added important information on the long-term safety and efficacy of this approach. Cladribine tablets (CladT) is an IRT given as two annual short courses of treatment, following which a majority of patients then demonstrate no significant MS disease activity over a period of years. Whether, and how, to treat patients beyond the first two years of treatment remains a matter for debate, as clinical evidence accumulates. We, a group of neurologists who manage people with RMS in Qatar, provide our expert consensus recommendations on the application and long-term management of CladT therapy based on our experience with treatment in the last 5 years. These include pragmatic recommendations for people with MS disease activity in years 3 and 4 (ie up to four years following first dose of CladT), and for people with or without MS disease activity in subsequent years. We believe our recommendations will help to ensure the optimal application of CladT-based IRT, with the potential benefit for the patient of achieving prolonged periods free of both MS disease symptoms and the burden of regular applications of immunosuppressive DMT.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10725680/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138806901","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Neuroprotective Effects of Leptin on the APP/PS1 Alzheimer's Disease Mouse Model: Role of Microglial and Neuroinflammation. 瘦素对APP/PS1阿尔茨海默病小鼠模型的神经保护作用:小胶质细胞和神经炎症的作用。
Degenerative neurological and neuromuscular disease Pub Date : 2023-10-25 eCollection Date: 2023-01-01 DOI: 10.2147/DNND.S427781
Jing Ma, Yi-Hui Hou, Zhe-Yan Liao, Zheng Ma, Xiao-Xuan Zhang, Jian-Li Wang, Yun-Bo Zhu, Hai-Lei Shan, Ping-Yue Wang, Cheng-Bo Li, Ying-Lei Lv, Yi-Lan Wei, Jie-Zhi Dou
{"title":"Neuroprotective Effects of Leptin on the APP/PS1 Alzheimer's Disease Mouse Model: Role of Microglial and Neuroinflammation.","authors":"Jing Ma,&nbsp;Yi-Hui Hou,&nbsp;Zhe-Yan Liao,&nbsp;Zheng Ma,&nbsp;Xiao-Xuan Zhang,&nbsp;Jian-Li Wang,&nbsp;Yun-Bo Zhu,&nbsp;Hai-Lei Shan,&nbsp;Ping-Yue Wang,&nbsp;Cheng-Bo Li,&nbsp;Ying-Lei Lv,&nbsp;Yi-Lan Wei,&nbsp;Jie-Zhi Dou","doi":"10.2147/DNND.S427781","DOIUrl":"10.2147/DNND.S427781","url":null,"abstract":"<p><strong>Background: </strong>Microglia are closely linked to Alzheimer's disease (AD) many years ago; however, the pathological mechanisms of AD remain unclear. The purpose of this study was to determine whether leptin affected microglia in the hippocampus of young and aged male APP/PS1 mice.</p><p><strong>Objective: </strong>In a transgenic model of AD, we investigated the association between intraperitoneal injection of leptin and microglia.</p><p><strong>Methods: </strong>We intraperitoneal injection of leptin (1mg/kg) every day for one week and analyzed inflammatory markers in microglia in the hippocampus of adult (6 months) and aged (12 months) APP/PS1 mice.</p><p><strong>Results: </strong>In all leptin treatment group, the brain Aβ levels were decrease. We found increased levels of IL-1β, IL-6 and microglial activation in the hippocampus of adult mice. Using aged mice as an experimental model for chronic neuroinflammation and leptin resistance, the number of Iba-1+ microglia and the levels of IL-1β/IL-6 in the hippocampus were greatly increased as compared to the adult. But between the leptin treatment and un-treatment, there were no difference.</p><p><strong>Conclusion: </strong>Leptin signaling would regulate the activation of microglia and the release of inflammatory factors, but it is not the only underlying mechanism in the neuroprotective effects of AD pathogenesis.</p>","PeriodicalId":93972,"journal":{"name":"Degenerative neurological and neuromuscular disease","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10613410/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71416318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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