Neurodegenerative disease management最新文献

{"title":"Mapping the journey of patients and care partners living with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia: developing a framework for improvements in care.","authors":"Holly A Rutherford, Beth K Rush, Adam Smith, Erin Sullivan, Clarissa Martinez-Rubio, Ali Toumadj, Roberta La Piana, Cynthia Cassandro","doi":"10.1080/17582024.2024.2404378","DOIUrl":"10.1080/17582024.2024.2404378","url":null,"abstract":"<p><p><b>Aim:</b> To identify and raise awareness of healthcare service gaps for individuals with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).<b>Materials & methods:</b> An ALSP patient journey map from symptom onset throughout disease course was developed using existing literature, patient and clinician feedback from a structured workshop and community survey data regarding attitudes toward genetic testing.<b>Results:</b> ALSP diagnosis is frequently delayed due to low awareness of this rare condition and symptom overlap with more common neurological conditions. Multiple factors impact patients' decision-making regarding genetic testing for ALSP, symptom management and participation in research studies.<b>Conclusion:</b> These results highlight the challenges faced by individuals with ALSP and should support program development to improve patient care.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11524202/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372376","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":"Walking performance differs between people with multiple sclerosis who perform distinct types of exercise.","authors":"Kristin A Johnson, Victoria M Bandera, Manfred Diehl, Heather J Leach, Brett W Fling","doi":"10.1080/17582024.2024.2389037","DOIUrl":"10.1080/17582024.2024.2389037","url":null,"abstract":"<p><p><b>Aim:</b> To determine whether walking performance differed between people with multiple sclerosis (MS) who performed distinct types, volumes and intensities of exercise.<b>Materials & methods:</b> Forty-five people with relapsing-remitting MS performed two trials of the 2-min walk test, one at a preferred speed and another at a fast speed. Gait metrics were measured by wireless inertial sensors. Participants reported the type (aerobic, resistance), volume and intensity of exercise performed.<b>Results:</b> Walking speed reserve and gait variability were better in participants who performed combined aerobic and resistance exercises compared with those who performed aerobic-only exercise.<b>Conclusion:</b> Walking performance differs in people with mild MS disability based on the type and volume of exercise performed.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11457625/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142000427","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":"Current and future applications of local field potential-guided programming for Parkinson's disease with the Percept™ rechargeable neurostimulator.","authors":"Weerawat Saengphatrachai, Joohi Jimenez-Shahed","doi":"10.1080/17582024.2024.2404386","DOIUrl":"10.1080/17582024.2024.2404386","url":null,"abstract":"<p><p>Deep brain stimulation (DBS) has been established as an effective neuromodulatory treatment for Parkinson's disease (PD) with motor complications or refractory tremor. Various DBS devices with unique technology platforms are commercially available and deliver continuous, open-loop stimulation. The Percept™ family of neurostimulators use BrainSense™ technology with five key features to sense local field potentials while stimulating, enabling integration of physiologic data into the routine practice of DBS programming. The newly approved Percept™ rechargeable RC implantable pulse generator offers a smaller, thinner design and reduced recharge time with prolonged recharge interval. In this review, we describe the application of local field potential sensing-based programming in PD and highlight the potential future clinical implementation of closed-loop stimulation using the Percept™ RC implantable pulse generator.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11524207/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142350678","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":"Bone marrow mesenchymal stem cell-derived extracellular vesicle infusion for amyotrophic lateral sclerosis.","authors":"Joshua J Crose, Arezou Crose, John T Ransom, Amy L Lightner","doi":"10.1080/17582024.2024.2344396","DOIUrl":"10.1080/17582024.2024.2344396","url":null,"abstract":"<p><p><b>Background:</b> In this pilot safety study, we hypothesized that a human bone marrow stem cell-derived extracellular vesicle (hBM-MSC EV) investigational product (IP) would be safe and exhibit potential efficacy in amyotrophic lateral sclerosis (ALS) patients.<b>Methods:</b> Ten ALS patients received two 10-ml intravenous infusions of the IP given 1 month apart and evaluated over 3 months.<b>Results:</b> There were no serious adverse events or adverse events related to the IP and 30% of subjects' ALS functional rating scale-revised (ALSFRS-R) scores did not decline.<b>Conclusion:</b> HBM-MSC EVs appear safe in ALS patients. This early investigation suggests a controlled study of EVs for the treatment of ALS is warranted.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11457643/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142350680","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":"Tofersen for SOD1 ALS.","authors":"William H Everett, Robert C Bucelli","doi":"10.1080/17582024.2024.2402216","DOIUrl":"10.1080/17582024.2024.2402216","url":null,"abstract":"<p><p>Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition affecting the motor system. The heterogenous nature of ALS complicates trial design. Genetic forms of ALS present an opportunity to intervene in a less heterogeneous population. ALS associated with gain of function mutations in <i>SOD1</i> make 'knock-down' strategies an attractive therapeutic approach. Tofersen, an antisense oligonucleotide that reduces expression of SOD1 via RNAase mediated degradation of <i>SOD1</i> mRNA, has shown robust effects on ALS biomarkers. While a Phase III trial of tofersen failed to meet its primary end point, open label extension data suggests that tofersen slows progression of SOD1 ALS.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11524200/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142350679","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":"Impact of a keto diet on symptoms of Parkinson's disease, biomarkers, depression, anxiety and quality of life: a longitudinal study.","authors":"Melanie M Tidman, Dawn Reid White, Tim A White","doi":"10.1080/17582024.2024.2352394","DOIUrl":"10.1080/17582024.2024.2352394","url":null,"abstract":"<p><p><b>Aim:</b> Evidence suggests low-carbohydrate diets (LCHF) may assist in treating neurodegenerative diseases such as Parkinson's disease (PD); however, gaps exist in the literature.<b>Patients & methods:</b> We conducted a small 24-week pilot study to investigate the effects of an LCHF diet on motor and nonmotor symptoms, health biomarkers, anxiety, and depression in seven people with PD. We also captured patient experiences during the process (quality of life [QoL]).<b>Results:</b> Participants reported improved biomarkers, enhanced cognition, mood, motor and nonmotor symptoms, and reduced pain and anxiety. Participants felt improvements enhanced their QoL.<b>Conclusion:</b> We conclude that an LCHF intervention is safe, feasible, and potentially effective in mitigating the symptoms of this disorder. However, more extensive randomized controlled studies are needed to create generalizable recommendations.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11457624/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141311267","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":"Comparison of time to clinically meaningful improvement in quality of life in neurological disorders in patients treated with natalizumab versus ocrelizumab.","authors":"Carrie M Hersh, Menglan Pang, Deborah M Miller, Marisa P McGinley, Megan Hyland, Tjalf Ziemssen, Robin L Avila","doi":"10.2217/nmt-2023-0047","DOIUrl":"10.2217/nmt-2023-0047","url":null,"abstract":"<p><p><b>Aim:</b> To assess time to improvement in Quality of Life in Neurological Disorders (Neuro-QoL) domains for patients treated with natalizumab versus ocrelizumab. <b>Methods:</b> Patients enrolled in the MS PATHS network who initiated treatment with either natalizumab or ocrelizumab rated the Neuro-QoL domains of physical function, symptoms, emotional health, cognitive function and social ability. <b>Results:</b> Time to clinically meaningful improvement was significantly shorter with natalizumab versus ocrelizumab for cognitive function (event time ratio [95% CI]: 0.37 [0.24-0.57]; p < 0.001), sleep disturbance (0.45 [0.28-0.72]; p = 0.001), social role participation (0.37 [0.21-0.66]; p = 0.001) and social role satisfaction (0.5 [0.31-0.8]; p = 0.004). <b>Conclusion:</b> Natalizumab had shorter time to clinically meaningful improvement in cognitive, sleep, and social role Neuro-QoL domains versus ocrelizumab.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140860977","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":"Comparing balance using the BESTest in Alzheimer, Huntington and Parkinson disease.","authors":"Lauren E Tueth, Ryan P Duncan, Beth E Crowner, Gammon M Earhart","doi":"10.1080/17582024.2024.2388507","DOIUrl":"10.1080/17582024.2024.2388507","url":null,"abstract":"<p><p><b>Aim:</b> Individuals with Alzheimer disease (AD), Huntington disease (HD) and Parkinson disease (PD) have impaired balance, and comparing these deficits could improve management of neurological diseases.<b>Methods:</b> Scores on the Balance Evaluation Systems Test (BESTest) were compared across three groups, consisting of individuals with AD, HD and PD in early stages of their respective disease.<b>Results:</b> Individuals with PD had significantly higher scores on the BESTest than individuals with AD (95% CI [4.30, 21.37], <i>p</i> < 0.01) or HD (95% CI [6.53, 24.18], <i>p</i> < 0.001). Individuals with AD and HD were not significantly different on the overall BESTest or any of its subsections.<b>Conclusion:</b> AD and HD may have overlapping pathologies resulting in early and similar balance impairments in these groups.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11457608/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142000425","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":"People with multiple sclerosis help design a tool to measure physical functioning and how it affects their daily lives: a plain language summary.","authors":"Paul Kamudoni, Dagmar Amtmann, Jeffrey Johns, Karon F Cook, Rana Salem, Sam Salek, Jana Raab, Rod Middleton, Pavle Repovic, Kevin N Alschuler, Gloria von Geldern, Annette Wundes, Amy Barrett, Oyebimpe Olayinka-Amao, Christian Henke","doi":"10.1080/17582024.2024.2357002","DOIUrl":"10.1080/17582024.2024.2357002","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This summary describes how researchers worked with people with multiple sclerosis (MS), neurologists and measurement experts to create an easy-to-use questionnaire to measure the physical function of people with MS. This questionnaire covers topics that are relevant and important to people with MS and their doctors.The ability to do what you want to do, when you want to do it, is one of the most important concerns for people with MS. This questionnaire could help doctors to record and manage how much MS affects people's lives.MS can bring a range of challenging symptoms such as '<b>brain fog</b>', tiredness, and problems with movement and balance. Many of these symptoms can make day-to-day activities, like working, very difficult for people with MS. Doctors currently use examinations like the <b>Expanded Disability Status Scale (EDSS)</b> and the <b>MS Functional Composite (MSFC)</b>, but these do not fully consider what is important to people living with MS. A questionnaire that specifically measures <b>physical functioning</b> of people with MS could help doctors and people with MS to better understand, communicate and manage the physical effects of MS. In this study, people with MS were asked to help create a questionnaire about physical function that reflects topics that are important to them.</p><p><strong>What were the results?: </strong>The PROMIS^®nq physical function - Multiple Sclerosis 15a (the PROMIS^® PF MS questionnaire) was successfully created with the help of people with MS. People with MS thought that the PROMIS^® PF MS questionnaire covered issues important to their physical function. Scores were in line with results of other physical symptom measurement scales like the EDSS.</p><p><strong>What do the results mean?: </strong>The PROMIS^® PF MS questionnaire could be used to meaningfully record physical function among people with MS.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11526672/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141760038","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":"A discussion with Philippe Huot: the challenges of discovering novel therapies for the treatment of Parkinson's disease.","authors":"Philippe Huot","doi":"10.1080/17582024.2024.2389034","DOIUrl":"10.1080/17582024.2024.2389034","url":null,"abstract":"","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11526725/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004871","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}