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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 Q3 Medicine 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

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.

肢带性肌营养不良症(LGMDs)是一种主要的肌肉疾病。治疗是迫切需要的,目前正在扩大基于安全性和有效性,采用单剂量基因治疗的原则,治疗单基因常染色体隐性遗传病。基因治疗已经在LGMD方面取得了进展,本文综述了在这些疾病方面取得的进展。本文首先介绍了lgmd的定义和分类背景。主要工作集中在LGMD基因治疗的进展,从实验研究到临床试验。所讨论的疾病包括LGMDs,其中做了最多的工作,包括calpainopathies (LGMD2A/R1), dysferlinopathies (LGMD2B/R2)和sarcoglycanopathy (LGMD2C/R5, LGMD2D/R3, LGMD2E/R4)。临床试验的早期成功为推动该领域的发展提供了一个模板,并有可能应用CRISPR/Cas9等新兴技术,这些技术可能会扩大基因治疗应用于患者护理的范围和功效。
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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 Q3 Medicine Pub Date : 2021-10-01 Epub Date: 2021-09-27 DOI: 10.2217/nmt-2021-0013
Gashirai K Mbizvo, Andrew J Larner

Aim: 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. Materials & methods: 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). Results: 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. Conclusion: If cognitive screeners producing categorical or continuous measures are dichotomized, calculated AUC may be an underestimate, thus affecting screening test accuracy.

目的:研究受试者工作特征图和曲线下面积(AUC)值是否可能在评估认知筛查工具作为二元预测因子而不是分类或连续量表时产生误导。材料和方法:AUC使用不同的方法(秩和、诊断优势比)计算,使用来自两个认知状态二元分类器(鼓掌标志、参加标志)、产生分类数据的筛选器(Codex)和连续量表筛选测试(Mini-Addenbrooke's cognitive Examination)的测试准确性研究数据。结果:对于所有筛查者,诊断优势比法计算的AUC均大于秩和法。当Codex和Mini-Addenbrooke认知检查作为二元(单一固定阈值)测试进行分析时,使用秩和方法的AUC分别低于筛选者作为分类或连续量表进行分析时的AUC。结论:如果对产生分类或连续测量的认知筛选者进行二分类,计算出的AUC可能会被低估,从而影响筛选测试的准确性。
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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 Q3 Medicine 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
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.
视神经脊髓炎视谱障碍(NMOSD)是一种罕见的自身免疫性疾病,以复发性视神经炎和横向脊髓炎为特征,常导致严重的残疾。抗aqp4免疫球蛋白G (IgG)是cd19阳性浆细胞的致病性产物,存在于大多数(但不是全部)NMOSD患者中,并与免疫介导的神经损伤有关。Inebilizumab是一种聚焦的人源IgG1κ抗cd19单克隆抗体,可靶向表达cd19的致病性B细胞。在一项II/III期试验中,与安慰剂相比,inebilizumab显著降低了NMOSD发作的参与者比例,并且耐受性良好。与接受安慰剂治疗的参与者相比,接受治疗的参与者残疾恶化的情况较少。Inebilizumab于2020年被美国FDA批准用于治疗抗aqp4抗体阳性的NMOSD。
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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 Q3 Medicine Pub Date : 2021-10-01 Epub Date: 2021-09-16 DOI: 10.2217/nmt-2021-0014
Aaron E Miller

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.

泰瑞氟米特(Teriflunomide)是一种每日一次的口腔疾病改善疗法,对于复发型多发性硬化症(MS)患者和首次临床发作提示多发性硬化症的患者,治疗时间长达12年,已证明其疗效、安全性和耐受性一致。该综述是对先前版本的更新,该版本检查了特立氟米特核心临床开发计划和扩展研究的数据。此后,从其他疾病改善疗法的积极比较试验、治疗相关的脑容量变化(使用萎缩归一化的结构图像评估分析)和包括患者报告的结果在内的真实证据中获得了数据。还介绍了特立氟米特对多发性硬化症患者潜在抗病毒作用的初步数据,包括感染2019年新型冠状病毒(SARS-CoV-2)患者的病例报告。
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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 Q3 Medicine Pub Date : 2021-10-01 Epub Date: 2021-08-19 DOI: 10.2217/nmt-2021-0011
Lauren Elizabeth Tueth, Ryan P Duncan

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.

帕金森病(PD)患者的肌肉骨骼(MSK)疼痛发生率高于年龄匹配的对照组。在本综述中,我们概述了有关帕金森病 MSK 疼痛的已知情况,重点关注颈部、肩部、膝部、髋部和腰部。我们还将对帕金森病患者的 MSK 疼痛的了解与未患帕金森病的老年人的了解进行了比较。最后,我们概述了与帕金森病患者 MSK 疼痛相关的未来研究领域。
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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 Q3 Medicine 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

Aim: The objective was to determine the prevalence of the potentially inappropriate antipsychotics and anticholinergics used in patients with Parkinson's disease. Materials & methods: 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. Results: 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. Conclusion: Older age and associated comorbidities, especially psychiatric and neurological comorbidities, increase the likelihood of patients with Parkinson's disease being prescribed antipsychotics and anticholinergics.

目的:目的是确定帕金森病患者使用的潜在不适当的抗精神病药物和抗胆碱能药物的患病率。材料与方法:一项横断面研究确定了抗精神病药物、抗胆碱能药物和治疗帕金森病的药物的处方。评估了抗胆碱能负荷,喹硫平和氯氮平被认为是适当的抗精神病药物。结果:共发现2965例帕金森病患者。精神疾病和其他神经系统疾病的存在与接受潜在不适当的抗精神病药物处方的更大可能性相关。合并症的数量越多,接受抗胆碱能药物治疗的可能性越大。结论:老年和相关的合并症,特别是精神和神经系统合并症,增加了帕金森病患者服用抗精神病药物和抗胆碱能药物的可能性。
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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 Q3 Medicine 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

Aim: 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. Patients & methods: An online survey comprising 42 questions was conducted on ALS patients during the peak of the COVID-19 pandemic. Results: 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. Conclusion: 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.

目的:了解马来西亚肌萎缩性侧索硬化症(ALS)患者对疾病的认知、管理以及2019冠状病毒病大流行对ALS患者的影响。患者和方法:在COVID-19大流行高峰期对ALS患者进行了一项包含42个问题的在线调查。结果:60名ALS患者中有37名(62%)直接或通过他们的照顾者回复。在2019冠状病毒病大流行期间,三分之二的患者受到医院预约、康复疗程突然中断和社交活动减少的负面影响。结论:这项研究提供了洞察患者的看法,他们的护理和管理的ALS在马来西亚,这将有助于实施变化,可以改善护理的人生活与这种毁灭性的疾病。
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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 Q3 Medicine 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

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.

对轻度至中度阿尔茨海默病的实际标准治疗只针对其症状。Masitinib是一种有效的选择性苯氨基噻唑型酪氨酸激酶抑制剂,目前正在进行治疗阿尔茨海默病(AD)的III期研究,目的是改变其进化,并具有多种药理靶点,如抑制肥大细胞活性,抑制小胶质细胞活化,调节a β和Tau蛋白信号通路以及预防突触损伤。在这里,我们回顾了研究马西替尼单药治疗AD的临床前和临床研究。所有的研究都表明,该药对阿尔茨海默病患者的认知功能有积极的影响,总体上具有良好的安全性和耐受性。
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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 Q3 Medicine 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

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.

术中神经生理信息可提高脑深部电刺激(DBS)导联定位的准确性。随后,DBS治疗可以通过特异性靶向病理活性来优化。在帕金森病中,β带(13-35 Hz)过度同步的局部场电位(LFPs)与动力学刚性症状及其对DBS治疗的反应相关,特别是低β带抑制(13-20 Hz)和高频γ促进(35-250 Hz)。在肌张力障碍中,lfp在theta/alpha (4- 13hz)、beta和gamma (60- 90hz)波段异常同步。相性肌张力障碍症状及其对DBS的反应与θ / α同步变化相关。在特发性震颤中,lfp在θ / α和β波段过度同步。自适应DBS系统将个性化神经生理信号的病理特征,自动传递特定时空参数的治疗性DBS脉冲。
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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 Q3 Medicine 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

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.

多发性硬化症(MS)持续的神经炎症和神经退行性病理导致身体和认知障碍的不可逆积累。可靠的MS疾病过程早期检测有助于MS患者的诊断、监测和治疗管理。最近的分析技术进步现在允许可靠的定量血清为基础的神经丝轻链(sNfL)水平,它提供了关于神经轴突损伤程度的时间信息。本文综述了sNfL与临床和认知结局、其他临床旁措施和治疗反应的关系和预测价值。sNfL测量是一种新兴的、无创的和疾病反应性的MS生物标志物,目前用于研究和临床试验环境。了解sNfL混杂因素和进一步的检测标准化将允许该生物标志物的临床应用。
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引用次数: 4
期刊
Neurodegenerative disease management
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