Jacob Sama, Wasi Khatri, Nathan Markus, Noel Dasgupta
{"title":"A review of Eplontersen use in hereditary transthyretin amyloidosis.","authors":"Jacob Sama, Wasi Khatri, Nathan Markus, Noel Dasgupta","doi":"10.1080/17582024.2025.2554385","DOIUrl":"https://doi.org/10.1080/17582024.2025.2554385","url":null,"abstract":"<p><p>Hereditary transthyretin amyloidosis is a progressive life-threatening disease characterized by deposition of abnormally folded amyloid fibrils into tissues leading to polyneuropathy and cardiomyopathy. Eplontersen, an antisense oligonucleotide has been FDA approved for the treatment of hereditary transthyretin amyloidosis with polyneuropathy in the United States. Eplontersen inhibits the translation of both variant and wildtype transthyretin in the liver, thereby preventing deposition in tissues. In the pivotal Phase III NEURO-TTRansform trial, Eplontersen significantly lowered serum transthyretin concentrations, improving neuropathic impairment and quality of life. Eplontersen was generally well tolerated in the treatment group, with the primary safety effects not significantly different from the control group. Eplontersen is suitable for long term use in patients with disease related polyneuropathy and is currently being studied for use in patients with cardiomyopathy. In this review, we discuss the clinical efficacy, mechanism of action, pharmacology, tolerability, and social determinants of health affecting the use of Eplontersen.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"1-10"},"PeriodicalIF":3.4,"publicationDate":"2025-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962478","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}
Beliu García-Parra, Josep M Guiu, Mónica Povedano, Pilar Modamio
{"title":"Geographic distribution of amyotrophic lateral sclerosis-related genes: a systematic review.","authors":"Beliu García-Parra, Josep M Guiu, Mónica Povedano, Pilar Modamio","doi":"10.1080/17582024.2025.2554382","DOIUrl":"https://doi.org/10.1080/17582024.2025.2554382","url":null,"abstract":"<p><strong>Introduction: </strong>Amyotrophic lateral sclerosis (ALS) is a rare motor neuron disease. There is no effective treatment, but disease-modifying therapies do exist. Objective. To identify the geographical distribution of ALS-related genes.</p><p><strong>Methods: </strong>A systematic review was conducted according to the PRISMA 2020 guidelines in PubMed and Web of Science. Inclusion criteria: patients with ALS, no age or gender restriction, English and Spanish languages, studies published until 31 July 2025.</p><p><strong>Results: </strong>Thirty-eight results were obtained, 32 were selected, 19 articles were assessed for eligibility, and 8 articles were included from databases. Three articles recommended by clinical experts were added, so 11 results were reviewed. This research showed that published articles on the geographic distribution of ALS-related genes are limited, particularly for underrepresented regions such as Africa.</p><p><strong>Conclusion: </strong>The findings demonstrate the need for intensified international research to improve knowledge of the genetic epidemiology of ALS.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"1-7"},"PeriodicalIF":3.4,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962475","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}
{"title":"Neuroprotective effects of ivermectin on Alzheimer's model induced by streptozotocin in rats.","authors":"Neda Farajpour, Hamid Soraya","doi":"10.1080/17582024.2025.2554374","DOIUrl":"https://doi.org/10.1080/17582024.2025.2554374","url":null,"abstract":"<p><strong>Background: </strong>Alzheimer's disease is a neurodegenerative condition characterized by memory deficits and cognitive decline. Ivermectin, an antiparasitic agent, has shown neuroprotective effects. The present study was conducted to determine the protective effects of ivermectin in a streptozocin-induced Alzheimer's model in rats.</p><p><strong>Methods: </strong>Alzheimer's model was induced by bilateral intracerebroventricular injection of streptozocin (3 mg/kg BW, 2 doses). Ivermectin was administered intraperitoneally at a dose of 2 mg/kg. On day 19, after behavioral memory and learning tests, the samples were collected for histological and enzymatic studies.</p><p><strong>Results: </strong>Ivermectin reduced the histopathological changes, including pyknotic and dead neurons and the accumulation of Aβ plaques. Ivermectin administration also reduced serum (<i>p</i> < 0.0001) and brain tissue (<i>p</i> < 0.01) acetylcholinesterase activity as well as improved learning (<i>p</i> < 0.05) and spatial memory (<i>p</i> < 0.0001).</p><p><strong>Conclusions: </strong>Ivermectin demonstrates protective effects in the STZ-induced Alzheimer's model by reducing pathological changes and Aβ plaques, acetylcholinesterase activity, as well as improving memory and learning.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"1-9"},"PeriodicalIF":3.4,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962472","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}
Isabel Portela Moreira, Paula Serrão, Lucinda Sequeira, Maria José Sá, Joana Guimarães, Maria Augusta Vieira-Coelho
{"title":"Cerebrospinal fluid catecholamines levels in Alzheimer's disease, frontotemporal dementia, and normal controls.","authors":"Isabel Portela Moreira, Paula Serrão, Lucinda Sequeira, Maria José Sá, Joana Guimarães, Maria Augusta Vieira-Coelho","doi":"10.1080/17582024.2025.2554527","DOIUrl":"https://doi.org/10.1080/17582024.2025.2554527","url":null,"abstract":"<p><strong>Aims: </strong>Catecholamine analysis and studies to modulate dopamine and norepinephrine in dementia have had contradictory results. We aimed to analyze the cerebrospinal fluid (CSF) levels of L-DOPA, dopamine, and norepinephrine of patients with Alzheimer's disease (AD), frontotemporal dementia (FTD) and normal controls (NC), and their relationship with clinical variables.</p><p><strong>Patients and methods: </strong>Cross-sectional, observational study, to analyze CSF L-DOPA, dopamine, and norepinephrine levels in 35 patients with AD, 16 with FTD, and 38 NC.</p><p><strong>Results: </strong>L-DOPA was significantly lower in AD than NC (AD vs NC <i>p</i> = 0.039, DFT vs NC <i>p</i> = 0.052, DFT vs DA <i>p</i> = 1.00). Norepinephrine was positively correlated with age (<i>r</i> = 0.343, <i>p</i> = 0.035). Dopamine was higher in participants under antidepressive medication (<i>p</i> = 0.022among all participants; <i>p</i> = 0.046 in NC).</p><p><strong>Conclusion: </strong>This is the first study reporting lower CSF L-DOPA levels in AD. Similar norepinephrine levels despite locus coeruleus neurons loss in AD point to compensatory mechanisms that could exacerbate L-DOPA deficiency.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"1-9"},"PeriodicalIF":3.4,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962481","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}
Tulia Fernanda Meira Garcia, Janaína Aparecida Favero Desio, Everton Ferreira de Souza, Silvana Fátima Costa Henkes, Luana Stangherlin Santos, Julcileia de Carvalho Muenho, Cinara Ludvig Gonçalves, Júlio César Claudino Dos Santos
{"title":"The silent saboteur: oxidative stress and the path to cognitive dysfunction.","authors":"Tulia Fernanda Meira Garcia, Janaína Aparecida Favero Desio, Everton Ferreira de Souza, Silvana Fátima Costa Henkes, Luana Stangherlin Santos, Julcileia de Carvalho Muenho, Cinara Ludvig Gonçalves, Júlio César Claudino Dos Santos","doi":"10.1080/17582024.2025.2510175","DOIUrl":"10.1080/17582024.2025.2510175","url":null,"abstract":"<p><p>Oxidative stress (OS) plays a central role in age-related cognitive decline and neurodegeneration and is increasingly recognized as a key factor in the pathogenesis of Alzheimer's disease (AD) and Parkinson's disease (PD). Elevated OS biomarkers are detectable from the earliest stages of these disorders. In this critical narrative review, we explore the bioenergetic cascade underlying neurodegeneration, emphasizing pathophysiological alterations, mechanisms, and therapeutic targets. Recent evidence suggests that OS and impaired cellular energy dynamics are both early markers and downstream effects of neuroinflammation, contributing to symptom severity and reduced treatment efficacy. A deeper understanding of these interrelated processes is essential for the development of more effective interventions. Monitoring OS-related metabolites may offer a promising strategy for identifying therapeutic targets and enabling early clinical intervention, ultimately aiming to reduce neuroinflammation and improve patient outcomes in AD and PD.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"121-148"},"PeriodicalIF":3.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12296065/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144160641","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}
{"title":"Challenges in managing comorbidities among people with dementia in low- and middle-income countries.","authors":"Manuella Zandoná, Raphael Machado Castilhos","doi":"10.1080/17582024.2025.2506337","DOIUrl":"10.1080/17582024.2025.2506337","url":null,"abstract":"","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"161-163"},"PeriodicalIF":3.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12296134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144079251","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}
{"title":"Characteristics of responders to interventions for Parkinson disease: a scoping systematic review.","authors":"Sidney T Baudendistel, Gammon M Earhart","doi":"10.1080/17582024.2025.2493465","DOIUrl":"10.1080/17582024.2025.2493465","url":null,"abstract":"<p><strong>Aims: </strong>The purpose of this review is to identify the characteristics of responders in interventions targeting motor function for individuals with Parkinson disease.</p><p><strong>Materials & methods: </strong>The primary search included, 'Parkinson' + 'responder.' A second, broader, search further included 'response' + 'responsiveness' + 'responsive.' Records were sorted by intervention: neuromodulation, pharmaceutical, physical, and placebo.</p><p><strong>Results: </strong>Thirteen studies were identified in the primary and 19 studies in the secondary search, culminating in 120 characteristics. For neuromodulation interventions, responders were younger at onset, more responsive to levodopa, and had more difficulties with activities of daily living. Responders to pharmaceuticals were younger at diagnosis. Physical intervention responders had worse balance, less balance confidence, and worse cognition. No relevant characteristics were identified for placebo interventions.</p><p><strong>Conclusions: </strong>Although there are clear limitations and gaps in the literature, responder analyses represent an important step toward more personalized treatments for the motor symptoms of Parkinson disease.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"173-186"},"PeriodicalIF":3.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12296146/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144012161","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}
Charlotte Massey, Esther Hobson, Alys Wyn Griffiths, Lucy Musson, Christopher McDermott
{"title":"Exploring mechanisms of behavior change for healthcare professionals in cough and secretion management in ALS.","authors":"Charlotte Massey, Esther Hobson, Alys Wyn Griffiths, Lucy Musson, Christopher McDermott","doi":"10.1080/17582024.2025.2506954","DOIUrl":"10.1080/17582024.2025.2506954","url":null,"abstract":"<p><strong>Objectives: </strong>To explore healthcare professionals' experiences managing cough and secretion problems in Amyotrophic Lateral Sclerosis (ALS).</p><p><strong>Methods: </strong>A qualitative study was completed with 23 individuals participating in four focus groups. Data was analyzed using reflexive thematic analysis and COM-B and theoretical domains framework (TDF) behavior change frameworks.</p><p><strong>Results: </strong>This study found that roles, responsibilities, and expectations needed to be clearly defined and that building relationships was important to support care delivery. Barriers identified included limited access to specialist care, equipment, and opportunities to gain knowledge and skills. A structured clinical assessment was highlighted to enable good-quality care. Data mapped most commonly to the environmental context/resources, knowledge, skills (TDF), and physical capability (COM-B) behavior change domains.</p><p><strong>Conclusion: </strong>Cough and secretion management in ALS is complex due to the multifaceted nature of the disease. This study emphasizes the need for future development of clinical interventions to support care.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":" ","pages":"149-160"},"PeriodicalIF":3.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12296054/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144102273","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}