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Evidence base for investigative and therapeutic modalities in chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy. 慢性炎症性脱髓鞘性多神经病变和多灶性运动神经病变的调查和治疗方法的证据基础。
IF 2.6
Neurodegenerative disease management Pub Date : 2022-02-01 Epub Date: 2022-01-10 DOI: 10.2217/nmt-2021-0015
Hendrik Stephan Goedee, Yusuf A Rajabally
{"title":"Evidence base for investigative and therapeutic modalities in chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy.","authors":"Hendrik Stephan Goedee,&nbsp;Yusuf A Rajabally","doi":"10.2217/nmt-2021-0015","DOIUrl":"https://doi.org/10.2217/nmt-2021-0015","url":null,"abstract":"<p><p>Chronic inflammatory demyelinating polyneuropathy, its variants and multifocal motor neuropathy belong to a spectrum of peripheral nerve disorders with complex dysimmune disease mechanisms. Awareness of the unique clinical phenotypes but also heterogeneity between patients is vital to arrive at early suspicion and ordering appropriate tests. This includes requirements for optimal electrodiagnostic protocol, aimed to capture sufficient electrophysiologic evidence for relevant abnormalities, a case-based approach on the eventual need to further expand the diagnostic armamentarium and correct reading of their results. Considerable phenotypical variation, diverse combinations of abnormalities found on diagnostic tests and heterogeneity in disease course and treatment response, all contribute to widespread differences in success rates on timely diagnosis and optimal treatment. We aim to provide a practical overview and guidance on relevant diagnostic and management strategies, including pitfalls and present a summary of the relevant novel developments in this field.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39665374","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
Corrigendum. 有待纠正。
IF 2.6
Neurodegenerative disease management Pub Date : 2022-02-01 Epub Date: 2022-01-10 DOI: 10.2217/nmt-2020-0021c1
{"title":"Corrigendum.","authors":"","doi":"10.2217/nmt-2020-0021c1","DOIUrl":"https://doi.org/10.2217/nmt-2020-0021c1","url":null,"abstract":"","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39665370","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
GOCOVRI® (amantadine) extended-release capsules in Parkinson's disease. GOCOVRI®(金刚烷胺)缓释胶囊治疗帕金森病
IF 2.6
Neurodegenerative disease management Pub Date : 2022-02-01 Epub Date: 2021-12-17 DOI: 10.2217/nmt-2021-0028
Thomas Müller
{"title":"GOCOVRI<sup>®</sup> (amantadine) extended-release capsules in Parkinson's disease.","authors":"Thomas Müller","doi":"10.2217/nmt-2021-0028","DOIUrl":"https://doi.org/10.2217/nmt-2021-0028","url":null,"abstract":"<p><p>Amantadine is an old, antiviral compound, which moderately improves motor behavior in Parkinson's disease. Its current resurgence results from an innovative, delayed uptake and extended release amantadine hydrochloride capsule, given at bedtime once daily. It is the only approved compound for reduction of involuntary movements, so called dyskinesia, in fluctuating orally levodopa treated patients. It additionally ameliorates 'OFF' intervals characterized by impaired motor behavior. These beneficial effects result from higher and more continuous brain delivery of amantadine. Future clinical research is warranted on preventive effects of this amantadine capsule combined with enzyme blockers of central monoamine oxidase B and peripheral catechol-O-methyltransferase on motor complications in orally levodopa treated patients, as all these pharmacological principles support the concept of continuous dopamine substitution.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39734057","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: 2
Side effects that occurred early in people with multiple sclerosis during the first year of treatment with cladribine tablets: a plain language summary. 多发性硬化症患者在使用克拉德滨片治疗的第一年早期发生的副作用:简单的语言总结。
IF 2.6
Neurodegenerative disease management Pub Date : 2022-02-01 Epub Date: 2022-01-12 DOI: 10.2217/nmt-2021-0041
Jiwon Oh, Bryan Walker, Gavin Giovannoni, Dominic Jack, Fernando Dangond, Axel Nolting, Julie Aldridge, Lori A Lebson, Thomas P Leist
{"title":"Side effects that occurred early in people with multiple sclerosis during the first year of treatment with cladribine tablets: a plain language summary.","authors":"Jiwon Oh,&nbsp;Bryan Walker,&nbsp;Gavin Giovannoni,&nbsp;Dominic Jack,&nbsp;Fernando Dangond,&nbsp;Axel Nolting,&nbsp;Julie Aldridge,&nbsp;Lori A Lebson,&nbsp;Thomas P Leist","doi":"10.2217/nmt-2021-0041","DOIUrl":"https://doi.org/10.2217/nmt-2021-0041","url":null,"abstract":"<p><p>People with multiple sclerosis (also shortened to MS) may have difficulties staying on treatment due to side effects. Cladribine tablets, approved for treating relapsing forms of MS, are given by mouth for four short periods over two years. The benefit of convenient dosing may be lost if side effects prevent people with MS from finishing their treatment. This is the summary of a study that examined side effects from cladribine tablets treatment in the first 12 weeks of two clinical studies called CLARITY and ORACLE-MS. Overall, 34.7% of participants who took cladribine tablets experienced drug-related side effects compared to 23.2% of participants who took placebo. Most side effects were mild and were seen in 54.8% of participants taking cladribine tablets and 59.1% taking the placebo. A low number of participants discontinued treatment due to side effects (1.6% of participants who took cladribine tablets; 1.4% of participants who took placebo). The researchers concluded that cladribine tablets are well-tolerated and people with MS are likely to complete the full treatment course. ClinicalTrials.gov NCT numbers: CLARITY study - NCT00213135 and ORACLE-MS study - NCT00725985.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39813239","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: 2
COVID-19-associated encephalitis or Creutzfeldt-Jakob disease: a case report. covid -19相关脑炎或克雅氏病1例报告
IF 2.6
Neurodegenerative disease management Pub Date : 2022-02-01 Epub Date: 2021-12-02 DOI: 10.2217/nmt-2021-0025
Gooya Tayyebi, Seyed Kazem Malakouti, Behnam Shariati, Leila Kamalzadeh
{"title":"COVID-19-associated encephalitis or Creutzfeldt-Jakob disease: a case report.","authors":"Gooya Tayyebi,&nbsp;Seyed Kazem Malakouti,&nbsp;Behnam Shariati,&nbsp;Leila Kamalzadeh","doi":"10.2217/nmt-2021-0025","DOIUrl":"https://doi.org/10.2217/nmt-2021-0025","url":null,"abstract":"<p><p><b>Background:</b> Accurate diagnosis and management of patients with rapidly progressive dementia may be challenging during the COVID-19 pandemic, which has negatively influenced the diagnostic performances, medical resource allocation and routine care for all non-COVID-19 diseases. <b>Case presentation:</b> We herein present a case of a 57-year-old male with rapidly progressive cognitive decline, headache, diplopia, myalgia, unsteady gait, aggression, depression, insomnia, hallucinations and delusions of persecution. COVID-19-associated encephalitis was briefly considered as a differential diagnosis. However, this hypothesis was rejected upon further investigation. A final diagnosis of sporadic Creutzfeldt-Jakob disease was made. <b>Conclusion:</b> A timely and accurate diagnosis of Creutzfeldt-Jakob disease gives patients and their families the chance to receive a good standard of healthcare and avoid extensive evaluations for other conditions.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8765092/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39951919","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: 8
MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design. MN-166(布司特)在肌萎缩性侧索硬化症的IIb/III期研究:COMBAT-ALS研究设计。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-12-01 Epub Date: 2021-11-24 DOI: 10.2217/nmt-2021-0042
Björn Oskarsson, Nicholas Maragakis, Richard S Bedlack, Namita Goyal, Jenny A Meyer, Angela Genge, Cynthia Bodkin, Samuel Maiser, Nathan Staff, Lorne Zinman, Nicholas Olney, John Turnbull, Benjamin Rix Brooks, Emelia Klonowski, Malath Makhay, Seiichi Yasui, Kazuko Matsuda
{"title":"MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design.","authors":"Björn Oskarsson,&nbsp;Nicholas Maragakis,&nbsp;Richard S Bedlack,&nbsp;Namita Goyal,&nbsp;Jenny A Meyer,&nbsp;Angela Genge,&nbsp;Cynthia Bodkin,&nbsp;Samuel Maiser,&nbsp;Nathan Staff,&nbsp;Lorne Zinman,&nbsp;Nicholas Olney,&nbsp;John Turnbull,&nbsp;Benjamin Rix Brooks,&nbsp;Emelia Klonowski,&nbsp;Malath Makhay,&nbsp;Seiichi Yasui,&nbsp;Kazuko Matsuda","doi":"10.2217/nmt-2021-0042","DOIUrl":"https://doi.org/10.2217/nmt-2021-0042","url":null,"abstract":"<p><p>Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with motor neuron loss as a defining feature. Despite significant effort, therapeutic breakthroughs have been modest. MN-166 (ibudilast) has demonstrated neuroprotective action by various mechanisms: inhibition of proinflammatory cytokines and macrophage migration inhibitory factor, phosphodiesterase inhibition, and attenuation of glial cell activation in models of ALS. Early-phase studies suggest that MN-166 may improve survival outcomes and slow disease progression in patients with ALS. This article describes the rationale and design of COMBAT-ALS, an ongoing randomized, double-blind, placebo-controlled, multicenter Phase IIb/III study in ALS. This study is designed to evaluate the pharmacokinetics, safety and tolerability and assess the efficacy of MN-166 on function, muscle strength, quality of life and survival in ALS.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39765803","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: 13
Repository corticotropin injection improves quality metrics in an observational study of multiple sclerosis relapse. 储存库促肾上腺皮质激素注射改善了多发性硬化症复发的观察性研究的质量指标。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-12-01 Epub Date: 2021-12-03 DOI: 10.2217/nmt-2021-0030
Jeffrey Kaplan, Tamara Miller, Matthew Baker, Bryan Due, Enxu Zhao
{"title":"Repository corticotropin injection improves quality metrics in an observational study of multiple sclerosis relapse.","authors":"Jeffrey Kaplan,&nbsp;Tamara Miller,&nbsp;Matthew Baker,&nbsp;Bryan Due,&nbsp;Enxu Zhao","doi":"10.2217/nmt-2021-0030","DOIUrl":"https://doi.org/10.2217/nmt-2021-0030","url":null,"abstract":"<p><p><b>Aim:</b> To determine whether clinicians evaluate American Academy of Neurology (AAN) quality metrics for patients with multiple sclerosis (MS) relapse and whether repository corticotropin injection (RCI) improves clinical and patient-reported outcomes associated with these metrics at 2 and 6 months after treatment. <b>Methods:</b> A multicenter, prospective, observational registry evaluating patients receiving RCI for MS relapse (N = 125) categorized data according to AAN quality metrics involving diagnosis, disability, fatigue, cognitive impairment, depression, and quality of life. <b>Results:</b> Clinicians assessed all 11 AAN quality metrics in patients with MS relapse. Disability, fatigue, cognitive impairment, depression, and quality of life outcomes improved with RCI therapy. <b>Conclusion:</b> RCI was associated with improved quality metrics, and AAN guidelines were followed during routine RCI treatment for MS relapse.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39688248","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: 2
Tongue measurements and pharyngeal residue in amyotrophic lateral sclerosis. 肌萎缩性侧索硬化症患者的舌头测量和咽残液。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-12-01 Epub Date: 2021-11-08 DOI: 10.2217/nmt-2021-0027
Milena Magalhães Augusto, Roberta Gonçalves da Silva, Mario Emílio Teixeira Dourado Júnior, Juliana Fernandes Godoy, Leonardo Wanderley Lopes, Leandro Pernambuco
{"title":"Tongue measurements and pharyngeal residue in amyotrophic lateral sclerosis.","authors":"Milena Magalhães Augusto,&nbsp;Roberta Gonçalves da Silva,&nbsp;Mario Emílio Teixeira Dourado Júnior,&nbsp;Juliana Fernandes Godoy,&nbsp;Leonardo Wanderley Lopes,&nbsp;Leandro Pernambuco","doi":"10.2217/nmt-2021-0027","DOIUrl":"https://doi.org/10.2217/nmt-2021-0027","url":null,"abstract":"<p><p><b>Aim:</b> We aimed to analyze the relationship between tongue measurements and vallecular residue in patients with amyotrophic lateral sclerosis (ALS). <b>Materials & methods:</b> Twenty-one patients with ALS were assessed for posterior maximum tongue isometric pressure (PMTIP) and posterior tongue isometric endurance (PTIE) by the Iowa Oral Performance Instrument; vallecular residue after 10 ml of moderately thickened consistency by Fiberoptic Endoscopic Evaluation of Swallowing; and tongue thickness (TT) by ultrasonography. <b>Results:</b> PMTIP, PTIE and TT were decreased compared with the reference values for healthy individuals and were not different between patients with and without vallecular residue. <b>Conclusion:</b> In ALS, PMTIP, PTIE and TT are not good predictors of vallecular residue in the tested volume and food consistency.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39599899","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: 2
Sporadic Huntington's disease in the Philippines: a case report. 菲律宾散发性亨廷顿舞蹈病1例报告。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-12-01 Epub Date: 2021-11-17 DOI: 10.2217/nmt-2021-0023
Laurence Kristoffer J Batino, John Hiyadan, Debbie Liquete, Manolo Flores
{"title":"Sporadic Huntington's disease in the Philippines: a case report.","authors":"Laurence Kristoffer J Batino,&nbsp;John Hiyadan,&nbsp;Debbie Liquete,&nbsp;Manolo Flores","doi":"10.2217/nmt-2021-0023","DOIUrl":"https://doi.org/10.2217/nmt-2021-0023","url":null,"abstract":"<p><p>Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder with core clinical features of choreoathetosis, cognitive deficits and behavioral changes. It is a rare disorder, primarily affecting the Caucasian population, and rarely Asians. To date, there are only two reported, genetically proven familial HD cases in the Philippines. We present the case of a 39-year-old Filipino male with a 10-year history of progressive behavior and personality changes followed by cognitive decline and choreoathetotic movements. Neuroimaging showed atrophy of both caudate and putamen with putaminal rim sign. Genetic testing revealed a 47 CAG trinucleotide repeats in the <i>Huntingtin</i> gene; family history is negative. This is the first, genetically proven, sporadic and the third HD case in the Philippines. Despite its rarity, this report highlights the importance of including HD as a possible cause of adult-onset chorea among Filipinos.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39631535","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
The remote assessment of parkinsonism supporting the ongoing development of interventions in Gaucher disease. 帕金森病的远程评估支持戈谢病干预措施的持续发展。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-12-01 Epub Date: 2021-10-20 DOI: 10.2217/nmt-2021-0032
Abigail Louise Higgins, Marco Toffoli, Stephen Mullin, Chiao-Yin Lee, Sofia Koletsi, Micol Avenali, Fabio Blandini, Anthony Hv Schapira
{"title":"The remote assessment of parkinsonism supporting the ongoing development of interventions in Gaucher disease.","authors":"Abigail Louise Higgins,&nbsp;Marco Toffoli,&nbsp;Stephen Mullin,&nbsp;Chiao-Yin Lee,&nbsp;Sofia Koletsi,&nbsp;Micol Avenali,&nbsp;Fabio Blandini,&nbsp;Anthony Hv Schapira","doi":"10.2217/nmt-2021-0032","DOIUrl":"https://doi.org/10.2217/nmt-2021-0032","url":null,"abstract":"<p><p>Mutations in <i>GBA</i> which are causative of Gaucher disease in their biallelic form, are the most common genetic risk factor for Parkinson's disease (PD). The diagnosis of PD relies upon clinically defined motor features which appear after irreversible neurodegeneration. Prodromal symptoms of PD may provide a means to predict latent pathology, years before the onset of motor features. Previous work has reported prodromal features of PD in <i>GBA</i> mutation carriers, however this has been insufficiently sensitive to identify those that will develop PD. The Remote Assessment of Parkinsonism Supporting Ongoing Development of Interventions in Gaucher Disease (RAPSODI GD) study assesses a large cohort of <i>GBA</i> mutation carriers, to aid development of procedures for earlier diagnosis of PD.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39531419","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: 2
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