Expert Review of Neurotherapeutics最新文献

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Cholesterol-modifying strategies for Alzheimer disease: promise or fallacy? 降低胆固醇治疗阿尔茨海默病的策略:希望还是谬误?
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-04-03 DOI: 10.1080/14737175.2025.2483928
Katia Azarfar, Boris Decourt, Brandon Sanchez Camacho, John Joshua Lawrence, Tania R Omondi, Marwan N Sabbagh
{"title":"Cholesterol-modifying strategies for Alzheimer disease: promise or fallacy?","authors":"Katia Azarfar, Boris Decourt, Brandon Sanchez Camacho, John Joshua Lawrence, Tania R Omondi, Marwan N Sabbagh","doi":"10.1080/14737175.2025.2483928","DOIUrl":"10.1080/14737175.2025.2483928","url":null,"abstract":"<p><strong>Introduction: </strong>As the world population ages, Alzheimer disease (AD) prevalence increases. However, understanding of AD etiology continues to evolve, and the pathophysiological processes involved are only partially elucidated. One compound suspected to play a role in the development and progression of AD is cholesterol. Several lines of evidence support this connection, yet it remains unclear whether cholesterol-modifying strategies have potential applications in the clinical management of AD.</p><p><strong>Areas covered: </strong>A deep literature search using PubMed was performed to prepare this narrative review. The literature search, performed in early 2024, was inclusive of literature from 1990 to 2024. After providing an overview of cholesterol metabolism, this study summarizes key preclinical studies that have investigated cholesterol-modifying therapies in laboratory models of AD. It also summarizes past and current clinical trials testing specific targets modulated by anti-cholesterol therapies in AD patients.</p><p><strong>Expert opinion: </strong>Based on current epidemiological and mechanistic studies, cholesterol likely plays a role in AD etiology. The use of cholesterol-modifying therapies could be a promising treatment approach if administered at presymptomatic to early AD phases, but it is unlikely to be efficient in mild, moderate, and late AD stages. Several recommendations are provided for hypercholesterolemia management in AD patients.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"521-535"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12068190/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143718345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cenobamate add-on therapy for drug-resistant focal seizures: a systematic review and meta-analysis. Cenobamate附加治疗耐药局灶性癫痫:一项系统回顾和荟萃分析。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-03-25 DOI: 10.1080/14737175.2025.2484439
Felipe Alves de Paiva, Artur Menegaz de Almeida, Aminah Abrão Fauaz Ritter Lima, Théo Jacovani Tozzo, Larissa Emi Tanimoto, Hamilton Roberto Moreira de Oliveira Carriço, Danilo Monteiro Ribeiro
{"title":"Cenobamate add-on therapy for drug-resistant focal seizures: a systematic review and meta-analysis.","authors":"Felipe Alves de Paiva, Artur Menegaz de Almeida, Aminah Abrão Fauaz Ritter Lima, Théo Jacovani Tozzo, Larissa Emi Tanimoto, Hamilton Roberto Moreira de Oliveira Carriço, Danilo Monteiro Ribeiro","doi":"10.1080/14737175.2025.2484439","DOIUrl":"10.1080/14737175.2025.2484439","url":null,"abstract":"<p><strong>Introduction: </strong>Cenobamate (CNB) is an anti-seizure medication (ASM) utilized for drug-resistant focal-onset seizures, which are difficult to manage with usual agents. Previous studies demonstrated that it can be effective in patients with refractory epilepsy.</p><p><strong>Methods: </strong>The MEDLINE, Cochrane, and Scopus databases were systematically searched up to 24 October 2024. A Random-effects model was employed to compute the Mean Difference (MD) and the Risk Ratio (RR) with 95% Confidence Intervals (CI). Statistical Analyses were performed utilizing RStudio 4.4.2.</p><p><strong>Results: </strong>Four studies were included, comprising 906 patients; 527 (59%) received CNB as add-on therapy. The results indicated that the 50% responder rate (RR 1.77; 95% CI: 1.28 to 2.44, <i>p</i> = 0.000551, I² = 70.3%) and seizure freedom (RR of 3.09; 95% CI: 1.91 to 5.00, <i>p</i> = 0.000004, I² = 8.7%) were significantly higher in this group.</p><p><strong>Conclusions: </strong>In this meta-analysis of four studies, CNB as an add-on therapy significantly reduced seizure frequency in patients with uncontrolled focal seizures, making it a promising option for improved seizure control and quality of life.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"591-597"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143700112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Two decades of metacognitive training for psychosis: successes, setbacks, and innovations. 二十年来治疗精神病的元认知训练:成功、挫折和创新。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-04-06 DOI: 10.1080/14737175.2025.2483204
Steffen Moritz, Ryan P Balzan, Mahesh Menon, Kim M Rojahn, Merle Schlechte, Ruth Veckenstedt, Daniel Schöttle, Antonia Meinhart
{"title":"Two decades of metacognitive training for psychosis: successes, setbacks, and innovations.","authors":"Steffen Moritz, Ryan P Balzan, Mahesh Menon, Kim M Rojahn, Merle Schlechte, Ruth Veckenstedt, Daniel Schöttle, Antonia Meinhart","doi":"10.1080/14737175.2025.2483204","DOIUrl":"10.1080/14737175.2025.2483204","url":null,"abstract":"<p><strong>Introduction: </strong>Schizophrenia is among the most debilitating mental health conditions. While antipsychotic medication represents the primary pillar of treatment, guidelines now also recommend psychotherapy. Metacognitive Training (MCT) has emerged over the past 20 years as a novel approach that addresses the cognitive biases involved in the pathogenesis of schizophrenia. MCT seeks to enhance patients' awareness of their cognitive distortions and reduce overconfidence. MCT is available in individual and group formats.</p><p><strong>Areas covered: </strong>This review provides a comprehensive overview of MCT, detailing its theoretical foundations, development, and implementation. The authors present meta-analyses demonstrating its efficacy in improving positive symptoms as well as negative symptoms and self-esteem. Lastly, the review covers the integration of the COGITO app to support MCT. For our narrative review we searched data bases including PubMed, Web of Science, EMBASE, PsycINFO, and MEDLINE.</p><p><strong>Expert opinion: </strong>MCT represents a significant advance in the treatment of schizophrenia, offering a flexible, low-threshold intervention that can be easily implemented in various clinical settings. The training's focus on metacognitive processes provides patients with tools to understand and manage their symptoms. Future research should seek to develop shortened as well as more personalized versions and investigate the long-term sustainability of the effects.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"579-590"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751812","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Managing impulse control and related behavioral disorders in Parkinson's disease: where we are in 2025? 管理帕金森病的冲动控制和相关行为障碍:2025年我们在哪里?
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-04-02 DOI: 10.1080/14737175.2025.2485337
Leonardo Rigon, Carmelo Fogliano, K Ray Chaudhuri, Karolina Poplawska-Domaszewicz, Cristian Falup-Pecurariu, Iulia Murasan, Mirjam Wolfschlag, Per Odin, Angelo Antonini
{"title":"Managing impulse control and related behavioral disorders in Parkinson's disease: where we are in 2025?","authors":"Leonardo Rigon, Carmelo Fogliano, K Ray Chaudhuri, Karolina Poplawska-Domaszewicz, Cristian Falup-Pecurariu, Iulia Murasan, Mirjam Wolfschlag, Per Odin, Angelo Antonini","doi":"10.1080/14737175.2025.2485337","DOIUrl":"10.1080/14737175.2025.2485337","url":null,"abstract":"<p><strong>Introduction: </strong>Impulse control and related behavioral disorders (ICBDs) commonly complicate Parkinson's disease (PD) course. The ICBDs spectrum encompasses two groups of conditions, with distinct pathophysiology: proper 'impulse control disorders (ICDs)' (e.g. gambling) and the 'ICDs related disorders (ICDs-RD)' (e.g. punding). Behavioral disturbances are associated with dopamine replacement therapies. ICBDs affect quality of life of patients and caregivers, making their management essential for reducing PD overall burden.</p><p><strong>Areas covered: </strong>This article reviews current management strategies for ICBDs in PD. The authors highlight strengths and limitations of these strategies, and explore the potential role of emerging treatment options, giving particular focus to new compounds and invasive therapies.</p><p><strong>Expert opinion: </strong>Prevention, close monitoring, and caregiver involvement are essential in managing ICBDs in PD. Treatment approaches should be tailored to ICBDs' functional impact and aimed to reduce the pulsatile stimulation of dopamine receptors, especially D2. Dopamine agonist (DA) tapering remains the primary therapeutic approach, alongside psychotherapy and second-line agents, like atypical antipsychotics and serotonin-noradrenaline reuptake inhibitors. Insights into ICDs pathophysiology and DA-specific pharmacodynamics indicate safer profiles for certain preparations (e.g. rotigotine patches) and possibly for D1/D5 agonists like tavapadon. Invasive treatments, including deep brain stimulation and infusion therapies, should be prioritized in advanced-stage PD complicated by ICBDs.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"537-554"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143735663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current perspectives on the recognition and management of treatment-resistant schizophrenia: challenges and opportunities. 目前对难治性精神分裂症的认识和管理:挑战和机遇。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-03-31 DOI: 10.1080/14737175.2025.2484434
Myrto Samara, Georgios Alevizopoulos, Vasilis P Bozikas, Ioannis Chatzimanolis, Dimitris Dikeos, Theodoros Mougiakos, Anastasia Nikolaou, Dimitrios Sakellariou, Charalampos Touloumis, Christos Tsopelas, Ofer Agid
{"title":"Current perspectives on the recognition and management of treatment-resistant schizophrenia: challenges and opportunities.","authors":"Myrto Samara, Georgios Alevizopoulos, Vasilis P Bozikas, Ioannis Chatzimanolis, Dimitris Dikeos, Theodoros Mougiakos, Anastasia Nikolaou, Dimitrios Sakellariou, Charalampos Touloumis, Christos Tsopelas, Ofer Agid","doi":"10.1080/14737175.2025.2484434","DOIUrl":"10.1080/14737175.2025.2484434","url":null,"abstract":"<p><strong>Introduction: </strong>Treatment-resistant schizophrenia (TRS) significantly impacts patients with schizophrenia, leading to a high disease burden, reduced quality of life, and functional impairment. Many patients fail to respond to standard antipsychotic treatments, requiring specialized therapeutic approaches. Clozapine remains the only approved treatment for patients with TRS, demonstrating effectiveness in reducing symptoms, hospitalizations, and risk of suicide. However, its use is often delayed due to concerns about adverse events, and the need for ongoing monitoring.</p><p><strong>Areas covered: </strong>This critical perspective incorporates insights from psychiatrists in Greece and a comprehensive literature analysis that includes clinical guidelines and systematic reviews. It highlights strategies for early diagnosis and timely initiation of clozapine, while emphasizing practical challenges in its use. Recommendations emphasize reducing treatment delays and overcoming barriers such as inadequate training and hesitancy among clinicians. A comprehensive literature search was conducted on PubMed, Google Scholar, and Cochrane Library without any date restrictions to ensure a thorough review of available evidence. The initial literature search was carried out in September 2024, with a subsequent search conducted in March 2025.</p><p><strong>Expert opinion: </strong>International guidelines consistently recommend clozapine as the first-line treatment for patients with TRS; nevertheless, the authors advocate enhanced awareness to optimize use. Most adverse events can be effectively managed with proper oversight, and early initiation is crucial to improving remission rates and the quality of life of patients with TRS. There is a need for systemic improvements in clinical practice, which requires evidence-based guidance to better address treatment efficacy in this challenging patient population.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"505-519"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Neuromodulation as a treatment strategy in Lennox-Gastaut syndrome: evidence and future directions. 神经调节作为lenox -胃综合征的治疗策略:证据和未来方向。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-03-02 DOI: 10.1080/14737175.2025.2474560
Gianluca D'Onofrio, Gianmichele Villano, Giovanni Dell'isola, Alberto Verrotti, Pasquale Striano
{"title":"Neuromodulation as a treatment strategy in Lennox-Gastaut syndrome: evidence and future directions.","authors":"Gianluca D'Onofrio, Gianmichele Villano, Giovanni Dell'isola, Alberto Verrotti, Pasquale Striano","doi":"10.1080/14737175.2025.2474560","DOIUrl":"10.1080/14737175.2025.2474560","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"501-504"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143531569","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The relevance of cortical thickness in migraine sufferers and implications to therapy. 偏头痛患者皮质厚度的相关性及其治疗意义。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-04-01 DOI: 10.1080/14737175.2025.2483924
Marcello Silvestro, Valentina Dortucci, Ilaria Orologio, Mario Cirillo, Fabrizio Esposito, Alessandro Tessitore, Antonio Russo
{"title":"The relevance of cortical thickness in migraine sufferers and implications to therapy.","authors":"Marcello Silvestro, Valentina Dortucci, Ilaria Orologio, Mario Cirillo, Fabrizio Esposito, Alessandro Tessitore, Antonio Russo","doi":"10.1080/14737175.2025.2483924","DOIUrl":"10.1080/14737175.2025.2483924","url":null,"abstract":"<p><strong>Introduction: </strong>Advanced neuroimaging studies have strongly contributed to clarify the gaps in the knowledge about migraine pathophysiology. Cortical thickness has garnered significant interest reflecting physiological processes such as gray matter neurogenesis and synaptic pruning, as well as pathophysiological mechanisms like neurodegeneration or plasticity changes associated with aging and disease. Evidence on cortical thickness highlights significant variability, likely due to migraine clinical complexity but also to methodological issues. Nevertheless, changes in the cortical thickness of areas involved in pain perception and modulation, as well as in cognitive and emotional attributes of pain experiences, have been consistently demonstrated reinforcing the concept of a dysfunctional neuro-limbic pain network in migraine.</p><p><strong>Areas covered: </strong>This review summarizes the available findings from advanced structural neuroimaging investigations, highlighting the most relevant findings and how they have contributed to the advancement in our understanding of migraine pathophysiology. This review is based on a literature search using PubMed along with the keyword 'migraine' combined with 'cortical thickness.'</p><p><strong>Expert opinion: </strong>Presently, it is challenging to ascertain whether the structural changes in migraine represent a primary phenomenon or the result of pain experience. Nevertheless, longitudinal neuroimaging studies have highlighted a role for treatments that, even if short-term, modulate cortical thickness, while also promoting the idea of structural changes as biomarkers.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"567-578"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751811","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current and emerging therapies for autoimmune encephalitis. 当前和新兴的自身免疫性脑炎治疗方法。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-05-01 Epub Date: 2025-03-26 DOI: 10.1080/14737175.2025.2483925
Darina Dinov, Linda Nguyen, Kyle Blackburn, Steven Vernino
{"title":"Current and emerging therapies for autoimmune encephalitis.","authors":"Darina Dinov, Linda Nguyen, Kyle Blackburn, Steven Vernino","doi":"10.1080/14737175.2025.2483925","DOIUrl":"10.1080/14737175.2025.2483925","url":null,"abstract":"<p><strong>Introduction: </strong>Autoimmune encephalitis (AIE) is an inflammatory neurological disorder often associated with autoantibodies targeting neural or glial antigens. Patients with AIE are often treated with immunotherapy, but multiple questions remain about the optimal treatment strategy for common AIE subtypes.</p><p><strong>Areas covered: </strong>The authors conducted a literature search of PubMed articles and Google Scholar articles using keywords 'autoimmune encephalitis,' 'anti-NMDA receptor encephalitis, 'LG1 encephalitis' from 2005 to 2024. This review briefly outlines the proposed pathophysiology of AIE with autoantibodies toward cell surface vs intracellular antigens. Next, the authors discuss treatments commonly used for AIE, and provide guidance on side effects and monitoring, and the evidence for treatment approaches for anti-NMDAr and LGI1 encephalitis is reviewed. In the final section, an overview of ongoing clinical trials and future therapies for AIE is provided.</p><p><strong>Expert opinion: </strong>Patients with AIE benefit from treatment with immunotherapy, but the evidence supporting specific treatment strategies is limited to observational studies. Successful clinical trials for AIE will provide new therapy options for patients, and the next generation of therapies may provide more targeted approaches to treating the condition.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"555-565"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143691356","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The utility of magnetoencephalography in epilepsy and its implications for treatment. 脑磁图在癫痫中的应用及其治疗意义。
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-04-01 Epub Date: 2025-02-22 DOI: 10.1080/14737175.2025.2470333
Samuel Molot-Toker, James T Rutka, Eisha Christian
{"title":"The utility of magnetoencephalography in epilepsy and its implications for treatment.","authors":"Samuel Molot-Toker, James T Rutka, Eisha Christian","doi":"10.1080/14737175.2025.2470333","DOIUrl":"10.1080/14737175.2025.2470333","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"375-379"},"PeriodicalIF":3.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476041","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Charcot-Marie-Tooth disease: a review of clinical developments and its management - What's new in 2025? 乳齿赘肉病:临床发展及其管理综述——2025年有什么新发现?
IF 3.4 2区 医学
Expert Review of Neurotherapeutics Pub Date : 2025-04-01 Epub Date: 2025-03-07 DOI: 10.1080/14737175.2025.2470980
Amedeo De Grado, Marina Serio, Paola Saveri, Chiara Pisciotta, Davide Pareyson
{"title":"Charcot-Marie-Tooth disease: a review of clinical developments and its management - What's new in 2025?","authors":"Amedeo De Grado, Marina Serio, Paola Saveri, Chiara Pisciotta, Davide Pareyson","doi":"10.1080/14737175.2025.2470980","DOIUrl":"10.1080/14737175.2025.2470980","url":null,"abstract":"<p><strong>Introduction: </strong>Charcot-Marie-Tooth disease (CMT) understanding and diagnostic rates are improving. Symptomatic management is still the only option, but many therapeutic approaches are under investigation, some in the clinical trial phase.</p><p><strong>Areas covered: </strong>Through a comprehensive search in PubMed, the ClinicalTrials.gov website, and the latest abstracts on the topic, the authors review the diagnostic advances and promising treatments, focusing on pharmacological and gene therapy/silencing approaches, and on clinical trial challenges. They also review current CMT management, including rehabilitation, orthotics, and associated symptoms and comorbidities.</p><p><strong>Expert opinion: </strong>The CMT field is evolving rapidly, with significant advances in genetic diagnosis and disease recognition. International networks and patient organization partnerships are vital for progress, enabling collaboration and large-scale studies. Metabolic neuropathies are relatively easier to target, and interim analysis results from the CMT-SORD trial suggest govorestat may become the first approved CMT drug. Gene therapy shows promise but currently faces safety and targeting challenges; PMP22 silencers for CMT1A are close to being tested in patients. New drugs, such as HDAC6 inhibitors, are also approaching the clinical trial phase, despite existing hurdles. Supportive care, including rehabilitation and orthotics, continues to improve quality of life. There is optimism that within the next decade, approved therapies will reduce disease burden.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"427-442"},"PeriodicalIF":3.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523037","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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