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Unmet needs and evolving treatment for limb girdle muscular dystrophies. 肢带肌营养不良症的未满足需求和不断发展的治疗方法。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-09-02 DOI: 10.2217/nmt-2020-0066
Eric Pozsgai, Danielle Griffin, Rachael Potter, Zarife Sahenk, Kelly Lehman, Louise R Rodino-Klapac, Jerry R Mendell
{"title":"Unmet needs and evolving treatment for limb girdle muscular dystrophies.","authors":"Eric Pozsgai,&nbsp;Danielle Griffin,&nbsp;Rachael Potter,&nbsp;Zarife Sahenk,&nbsp;Kelly Lehman,&nbsp;Louise R Rodino-Klapac,&nbsp;Jerry R Mendell","doi":"10.2217/nmt-2020-0066","DOIUrl":"https://doi.org/10.2217/nmt-2020-0066","url":null,"abstract":"<p><p>Limb-girdle muscular dystrophies (LGMDs) represent a major group of muscle disorders. Treatment is sorely needed and currently expanding based on safety and efficacy adopting principles of single-dosing gene therapy for monogenic autosomal recessive disorders. Gene therapy has made in-roads for LGMD and this review describes progress that has been achieved for these conditions. This review first provides a background on the definition and classification of LGMDs. The major effort focuses on progress in LGMD gene therapy, from experimental studies to clinical trials. The disorders discussed include the LGMDs where the most work has been done including calpainopathies (LGMD2A/R1), dysferlinopathies (LGMD2B/R2) and sarcoglycanopathies (LGMD2C/R5, LGMD2D/R3, LGMD2E/R4). Early success in clinical trials provides a template to move the field forward and potentially apply emerging technology like CRISPR/Cas9 that may enhance the scope and efficacy of gene therapy applied to patient care.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39376811","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: 21
Receiver operating characteristic plot and area under the curve with binary classifiers: pragmatic analysis of cognitive screening instruments. 二元分类器的受试者工作特征图和曲线下面积:认知筛选工具的语用分析。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-09-27 DOI: 10.2217/nmt-2021-0013
Gashirai K Mbizvo, Andrew J Larner
{"title":"Receiver operating characteristic plot and area under the curve with binary classifiers: pragmatic analysis of cognitive screening instruments.","authors":"Gashirai K Mbizvo,&nbsp;Andrew J Larner","doi":"10.2217/nmt-2021-0013","DOIUrl":"https://doi.org/10.2217/nmt-2021-0013","url":null,"abstract":"<p><p><b>Aim:</b> To examine whether receiver operating characteristic plots and area under the curve (AUC) values may be potentially misleading when assessing cognitive screening instruments as binary predictors rather than as categorical or continuous scales. <b>Materials & methods:</b> AUC was calculated using different methods (rank-sum, diagnostic odds ratio) using data from test accuracy studies of two binary classifiers of cognitive status (applause sign, attended with sign), a screener producing categorical data (Codex), and a continuous scale screening test (Mini-Addenbrooke's Cognitive Examination). <b>Results:</b> For all screeners, AUC calculated using diagnostic odds ratio method was greater than using rank-sum method. When Codex and Mini-Addenbrooke's Cognitive Examination were analyzed as binary (single fixed threshold) tests, AUC using rank-sum method was lower than when screeners were analyzed as categorical or continuous scales, respectively. <b>Conclusion:</b> If cognitive screeners producing categorical or continuous measures are dichotomized, calculated AUC may be an underestimate, thus affecting screening test accuracy.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39454363","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: 4
Inebilizumab for treatment of neuromyelitis optica spectrum disorder. 依比利珠单抗治疗视神经脊髓炎谱系障碍。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-09-06 DOI: 10.2217/nmt-2021-0017
Mark J Tullman, Aram Zabeti, Scott Vuocolo, Quinn Dinh
{"title":"Inebilizumab for treatment of neuromyelitis optica spectrum disorder.","authors":"Mark J Tullman,&nbsp;Aram Zabeti,&nbsp;Scott Vuocolo,&nbsp;Quinn Dinh","doi":"10.2217/nmt-2021-0017","DOIUrl":"https://doi.org/10.2217/nmt-2021-0017","url":null,"abstract":"Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease characterized by recurrent optic neuritis and transverse myelitis often resulting in severe disability. Anti-aquaporin-4-immunoglobulin (Ig) G is a pathogenic product of CD19-positive plasma cells found in most, but not all, individuals with NMOSD and is associated with immune-mediated neurologic injury. Inebilizumab, an afucosylated humanized IgG1 κ, anti-CD19 monoclonal antibody, may target pathogenic CD19-expressing B cells. In a Phase II/III trial, inebilizumab significantly reduced the proportion of participants experiencing an NMOSD attack and was well tolerated versus placebo. Fewer treated participants had worsening disability than those receiving placebo. Inebilizumab was approved in 2020 by the US FDA for treatment of anti-aquaporin-4 antibody positive NMOSD.","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39405026","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: 4
An updated review of teriflunomide's use in multiple sclerosis. 特立氟米特用于多发性硬化症的最新综述。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-09-16 DOI: 10.2217/nmt-2021-0014
Aaron E Miller
{"title":"An updated review of teriflunomide's use in multiple sclerosis.","authors":"Aaron E Miller","doi":"10.2217/nmt-2021-0014","DOIUrl":"https://doi.org/10.2217/nmt-2021-0014","url":null,"abstract":"<p><p>Teriflunomide, a once daily, oral disease-modifying therapy, has demonstrated consistent efficacy, safety and tolerability in patients with relapsing forms of multiple sclerosis (MS) and with a first clinical episode suggestive of MS treated up to 12 years. This review is an update to a previous version that examined data from the teriflunomide core clinical development program and extension studies. Data have since become available from active comparator trials with other disease-modifying therapies, treatment-related changes in brain volume (analyzed using structural image evaluation using normalization of atrophy) and real-world evidence including patient-reported outcomes. Initial data on the potential antiviral effects of teriflunomide in patients with MS, including case reports of patients infected with the 2019 novel coronavirus (SARS-CoV-2), are also presented.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39405029","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: 16
Musculoskeletal pain in Parkinson's disease: a narrative review. 帕金森病的肌肉骨骼疼痛：叙述性综述。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-08-19 DOI: 10.2217/nmt-2021-0011
Lauren Elizabeth Tueth, Ryan P Duncan
{"title":"Musculoskeletal pain in Parkinson's disease: a narrative review.","authors":"Lauren Elizabeth Tueth, Ryan P Duncan","doi":"10.2217/nmt-2021-0011","DOIUrl":"10.2217/nmt-2021-0011","url":null,"abstract":"<p><p>The prevalence of musculoskeletal (MSK) pain in people with Parkinson's disease (PD) is higher than that of age-matched controls. In this review, we outline what is known about MSK pain in PD, focusing on the neck, shoulder, knee, hip and low back. We also compare what is known about MSK pain in PD to what is known in older adults without PD. Finally, we outline areas of for future research related to MSK pain in people with PD.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8515213/pdf/nmt-11-373.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39327299","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
Potentially inappropriate prescriptions of antipsychotic and anticholinergic drugs in patients with Parkinson's disease. 帕金森病患者的抗精神病和抗胆碱能药物处方可能不适当。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-10-01 Epub Date: 2021-09-16 DOI: 10.2217/nmt-2021-0021
Luis Fernando Valladales-Restrepo, Nathalia Marcela Peña-Verjan, Katherine Vargas-Díaz, Jorge Enrique Machado-Alba
{"title":"Potentially inappropriate prescriptions of antipsychotic and anticholinergic drugs in patients with Parkinson's disease.","authors":"Luis Fernando Valladales-Restrepo,&nbsp;Nathalia Marcela Peña-Verjan,&nbsp;Katherine Vargas-Díaz,&nbsp;Jorge Enrique Machado-Alba","doi":"10.2217/nmt-2021-0021","DOIUrl":"https://doi.org/10.2217/nmt-2021-0021","url":null,"abstract":"<p><p><b>Aim:</b> The objective was to determine the prevalence of the potentially inappropriate antipsychotics and anticholinergics used in patients with Parkinson's disease. <b>Materials & methods:</b> A cross-sectional study identified the prescription of antipsychotics, anticholinergics and drugs for the treatment of Parkinson's disease. The anticholinergic burden was evaluated, and quetiapine and clozapine were considered to be adequate antipsychotics. <b>Results:</b> 2965 patients with Parkinson's disease were identified. The presence of psychiatric disorders and other neurological pathologies was associated with a greater probability of receiving potentially inappropriate antipsychotic prescriptions. The presence of greater number of comorbidities was associated with a greater probability of receiving anticholinergics. <b>Conclusion:</b> Older age and associated comorbidities, especially psychiatric and neurological comorbidities, increase the likelihood of patients with Parkinson's disease being prescribed antipsychotics and anticholinergics.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39418892","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
A survey on patients' disease perception and the impact of the COVID-19 pandemic on persons living with amyotrophic lateral sclerosis in Malaysia. 马来西亚肌萎缩性侧索硬化症患者疾病认知及新冠肺炎疫情对患者影响调查
IF 2.6
Neurodegenerative disease management Pub Date : 2021-08-01 Epub Date: 2021-07-21 DOI: 10.2217/nmt-2021-0004
Suzanna Edgar, Nur Adilah Abdul-Aziz, Ee Chin Loh, David Capelle, Khean-Jin Goh, Lydia Abdul Latif, Nortina Shahrizaila, Azlina Ahmad-Annuar
{"title":"A survey on patients' disease perception and the impact of the COVID-19 pandemic on persons living with amyotrophic lateral sclerosis in Malaysia.","authors":"Suzanna Edgar,&nbsp;Nur Adilah Abdul-Aziz,&nbsp;Ee Chin Loh,&nbsp;David Capelle,&nbsp;Khean-Jin Goh,&nbsp;Lydia Abdul Latif,&nbsp;Nortina Shahrizaila,&nbsp;Azlina Ahmad-Annuar","doi":"10.2217/nmt-2021-0004","DOIUrl":"https://doi.org/10.2217/nmt-2021-0004","url":null,"abstract":"<p><p><b>Aim:</b> To investigate the patients' perception of their disease, its management and the impact of the COVID-19 pandemic on persons living with amyotrophic lateral sclerosis (ALS) in Malaysia. <b>Patients & methods:</b> An online survey comprising 42 questions was conducted on ALS patients during the peak of the COVID-19 pandemic. <b>Results:</b> Responses were received from 37/60 (62%) participants with ALS directly or through their caregivers. During the COVID-19 pandemic, two-thirds of patients were negatively impacted by the sudden disruption to their hospital appointments, rehabilitation sessions and reduced social interactions. <b>Conclusion:</b> This study provided insight into patients' perception of their care and management of ALS in Malaysia which will facilitate in implementing changes that can improve care to persons living with this devastating illness.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8320654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39205769","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: 3
Masitinib for the treatment of Alzheimer's disease. 马西替尼用于治疗阿尔茨海默病。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-08-01 Epub Date: 2021-08-20 DOI: 10.2217/nmt-2021-0019
Miren Ettcheto, Amanda Cano, Elena Sanchez-López, Ester Verdaguer, Jaume Folch, Carme Auladell, Antoni Camins
{"title":"Masitinib for the treatment of Alzheimer's disease.","authors":"Miren Ettcheto,&nbsp;Amanda Cano,&nbsp;Elena Sanchez-López,&nbsp;Ester Verdaguer,&nbsp;Jaume Folch,&nbsp;Carme Auladell,&nbsp;Antoni Camins","doi":"10.2217/nmt-2021-0019","DOIUrl":"https://doi.org/10.2217/nmt-2021-0019","url":null,"abstract":"<p><p>The actual standard treatment for mild-to-moderately severe Alzheimer's disease only attacks its symptoms. Masitinib is a potent and selective phenylaminothiazole-type tyrosine kinase inhibitor which is currently in Phase III studies for the treatment of Alzheimer's disease (AD) with the aim of modifying its evolution and with multiple pharmacological targets such as inhibition of mast cells activity, inhibition of microglia activation, modulation of Aβ and Tau protein signaling pathway and prevention of synaptic damage. Here, we review the preclinical and clinical studies that investigated the administration of masitinib treatment in monotherapy in AD. All research studies revealed positive effects concerning the cognitive functions in AD and generally with good safety and tolerability.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39327163","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: 10
Neurophysiological biomarkers to optimize deep brain stimulation in movement disorders. 优化运动障碍深部脑刺激的神经生理生物标志物。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-08-01 Epub Date: 2021-07-15 DOI: 10.2217/nmt-2021-0002
Daniel Sirica, Angela L Hewitt, Christopher G Tarolli, Miriam T Weber, Carol Zimmerman, Aida Santiago, Andrew Wensel, Jonathan W Mink, Karlo J Lizárraga
{"title":"Neurophysiological biomarkers to optimize deep brain stimulation in movement disorders.","authors":"Daniel Sirica,&nbsp;Angela L Hewitt,&nbsp;Christopher G Tarolli,&nbsp;Miriam T Weber,&nbsp;Carol Zimmerman,&nbsp;Aida Santiago,&nbsp;Andrew Wensel,&nbsp;Jonathan W Mink,&nbsp;Karlo J Lizárraga","doi":"10.2217/nmt-2021-0002","DOIUrl":"https://doi.org/10.2217/nmt-2021-0002","url":null,"abstract":"<p><p>Intraoperative neurophysiological information could increase accuracy of surgical deep brain stimulation (DBS) lead placement. Subsequently, DBS therapy could be optimized by specifically targeting pathological activity. In Parkinson's disease, local field potentials (LFPs) excessively synchronized in the beta band (13-35 Hz) correlate with akinetic-rigid symptoms and their response to DBS therapy, particularly low beta band suppression (13-20 Hz) and high frequency gamma facilitation (35-250 Hz). In dystonia, LFPs abnormally synchronize in the theta/alpha (4-13 Hz), beta and gamma (60-90 Hz) bands. Phasic dystonic symptoms and their response to DBS correlate with changes in theta/alpha synchronization. In essential tremor, LFPs excessively synchronize in the theta/alpha and beta bands. Adaptive DBS systems will individualize pathological characteristics of neurophysiological signals to automatically deliver therapeutic DBS pulses of specific spatial and temporal parameters.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/af/a8/nmt-11-315.PMC8977945.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39183915","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: 11
Disease biomarkers in multiple sclerosis: current serum neurofilament light chain perspectives. 多发性硬化症的疾病生物标志物:当前血清神经丝轻链的观点。
IF 2.6
Neurodegenerative disease management Pub Date : 2021-08-01 Epub Date: 2021-07-01 DOI: 10.2217/nmt-2020-0058
Dejan Jakimovski, Michael G Dwyer, Niels Bergsland, Bianca Weinstock-Guttman, Robert Zivadinov
{"title":"Disease biomarkers in multiple sclerosis: current serum neurofilament light chain perspectives.","authors":"Dejan Jakimovski,&nbsp;Michael G Dwyer,&nbsp;Niels Bergsland,&nbsp;Bianca Weinstock-Guttman,&nbsp;Robert Zivadinov","doi":"10.2217/nmt-2020-0058","DOIUrl":"https://doi.org/10.2217/nmt-2020-0058","url":null,"abstract":"<p><p>The continuous neuroinflammatory and neurodegenerative pathology in multiple sclerosis (MS) results in irreversible accumulation of physical and cognitive disability. Reliable early detection of MS disease processes can aid in the diagnosis, monitoring and treatment management of MS patients. Recent assay technological advancements now allow reliable quantification of serum-based neurofilament light chain (sNfL) levels, which provide temporal information regarding the degree of neuroaxonal damage. The relationship and predictive value of sNfL with clinical and cognitive outcomes, other paraclinical measures and treatment response is reviewed. sNfL measurement is an emerging, noninvasive and disease-responsive MS biomarker that is currently utilized in research and clinical trial settings. Understanding sNfL confounders and further assay standardization will allow clinical implementation of this biomarker.</p>","PeriodicalId":19114,"journal":{"name":"Neurodegenerative disease management","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2021-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39149593","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: 4
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