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Movement Disorders Clinical Practice最新文献

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In Memoriam Kapil Sethi, M.D. (1953-2024). 悼念卡皮尔-塞西医学博士(1953-2024)。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-03 DOI: 10.1002/mdc3.14204
Matthew B Stern, Anthony Lang
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引用次数: 0
Juvenile Parkinsonism and Cognitive Impairment in a Patient with Compound Heterozygous Variants in the BTD Gene- an Unusual Presentation of Biotinidase Deficiency. 一名 BTD 基因复合杂合子变异体患者的幼年帕金森症和认知障碍--生物素酶缺乏症的异常表现。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-06-28 DOI: 10.1002/mdc3.14154
Baikuntha Panigrahi, Divya M Radhakrishnan, Ayush Agarwal, Roopa Rajan, Divyani Garg, Animesh Das, Awadh Kishor Pandit, Achal K Srivastava
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引用次数: 0
Automated Motion Sensor-Based Functional Mapping Improves Deep Brain Stimulation Programming Efficiency. 基于运动传感器的自动功能绘图提高了深部脑刺激编程效率
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-08-13 DOI: 10.1002/mdc3.14181
Vibhor Krishna, Aaron Hadley, Danielle Englert, Nicholas Fleming, Benjamin L Walter, Erica Hennigs, Aristide Merola, Dustin A Heldman
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引用次数: 0
Continuous Subcutaneous Foslevodopa-Foscarbidopa in Parkinson's Disease: A Mini-Review of Current Scope and Future Outlook. 帕金森病中的连续皮下注射磷左多巴-福斯卡比多巴:当前范围和未来展望微型综述》。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-07-11 DOI: 10.1002/mdc3.14161
Mickael Aubignat, Melissa Tir

Background: Parkinson's disease (PD) is a progressive neurodegenerative disorder characterized by motor and non-motor symptoms, primarily because of the impairment of dopaminergic neurons. Long-term use of levodopa, the standard PD treatment, often results in fluctuating therapeutic effects and dyskinesia, necessitating alternative therapies.

Objectives: This review aims to synthesize current insights and clinical experiences with foslevodopa-foscarbidopa, focusing on its pharmacokinetics, efficacy, and safety profile, to evaluate its potential in transforming PD therapy.

Methods: A systematic literature search was conducted up to November 2023 using databases PubMed, Web of Science, and Cochrane Library. The search yielded eight eligible articles, including pharmacological studies, case reports, observational studies, and controlled trials. No language restrictions were applied.

Results: Foslevodopa and foscarbidopa, as prodrugs of levodopa and carbidopa, exhibited excellent chemical stability and solubility, facilitating continuous subcutaneous infusion. Clinical trials demonstrated that these prodrugs maintain stable levodopa levels, thereby addressing the limitations of oral levodopa therapy. Phase 1 and 3 studies indicated significant improvements in motor function and quality of life in advanced PD patients. However, a higher incidence of treatment-emergent adverse events, mainly infusion site reactions, was observed compared to oral therapies.

Conclusions: Foslevodopa-foscarbidopa emerges as a promising alternative for advanced PD treatment, offering sustained symptom control. Its efficacy in managing motor fluctuations and dyskinesia makes it a viable option in the PD therapeutic spectrum. Future research should focus on long-term safety, economic impact, and broader accessibility. Foslevodopa-foscarbidopa is now commercially distributed in many countries in Europe and in Japan.

背景:帕金森病(PD)是一种进行性神经退行性疾病,主要由于多巴胺能神经元受损而导致运动和非运动症状。左旋多巴是治疗帕金森病的标准药物,但长期使用左旋多巴往往会导致疗效波动和运动障碍,因此有必要采用替代疗法:本综述旨在综合目前对磷左多巴-磷卡比多巴的认识和临床经验,重点关注其药代动力学、疗效和安全性,以评估其改变帕金森病疗法的潜力:使用 PubMed、Web of Science 和 Cochrane Library 等数据库对截至 2023 年 11 月的文献进行了系统检索。检索结果显示有 8 篇符合条件的文章,包括药理学研究、病例报告、观察性研究和对照试验。检索结果不限制语言:结果:作为左旋多巴和卡比多巴的原药,福斯氯多巴和福斯卡比多巴具有良好的化学稳定性和溶解性,便于持续皮下注射。临床试验表明,这些原药可保持左旋多巴水平的稳定,从而解决了口服左旋多巴疗法的局限性。1 期和 3 期研究表明,晚期帕金森病患者的运动功能和生活质量均有显著改善。然而,与口服疗法相比,治疗突发不良事件的发生率较高,主要是输液部位反应:结论:磷来伏多巴-福斯卡比多巴是治疗晚期帕金森病的一种很有前景的替代疗法,能持续控制症状。它在控制运动波动和运动障碍方面的疗效使其成为帕金森病治疗领域的一个可行选择。未来的研究应侧重于长期安全性、经济影响和更广泛的可及性。目前,磷左多巴-磷卡比多巴已在欧洲许多国家和日本上市销售。
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引用次数: 0
Vibrotactile Foot Device for Freezing of Gait in Parkinson's Disease: A Pilot Study. 振动触觉足部装置治疗帕金森病步态冻结:一项试点研究
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-08-02 DOI: 10.1002/mdc3.14177
Shanshan Cen, Jinghong Ma, Hong Sun, Hui Zhang, Yuan Li, Wei Mao, Erhe Xu, Shanshan Mei, Jagadish K Chhetri, Zheng Ruan, Hui Wang, Kailiang Wang, Piu Chan

Background: Vibrotactile stimulation has been studied in its efficacy of reducing freezing of gait (FOG) in patients with Parkinson's disease (PD). However, the results are still controversial. We evaluated the efficacy of a newly developed vibrotactile foot device on freezing severity and gait measures in PD patients with FOG.

Objective: To evaluate the efficacy of vibrotactile foot device on PD patients with FOG.

Methods: Thirty-three PD patients with FOG were examined during their "off" medication state. The efficacy of the vibrotactile foot device was evaluated using a gait protocol comprising walking trials with vibrotactile stimulation "off" and "on." Walking trials were videotaped for the offline rating by two movement disorder specialists. The Opal inertial sensor unit (128 Hz; Mobility Lab; APDM Inc., Portland, OR, USA) was used for quantitative gait analysis.

Results: The results demonstrated 33.1% reduction in number of FOG episodes (P < 0.001) and 32.6% reduction of freezing episodes (P < 0.001). Quantitative gait analysis showed a significant increase in step length (P = 0.033). A moderate negative correlation was observed between the change of percent time frozen and age (r = -0.415, P = 0.016). 73% of participants reported minimal to substantial improvement in walking with this vibrating stimulation delivered by the vibrotactile foot device.

Conclusions: The vibrotactile foot device is an efficient device that could significantly reduce freezing severity and provide gait regulation to patients with PD experiencing frequent freezing. It could potentially be used in the home environment for improving the quality of life.

背景:振动触觉刺激对减少帕金森病(PD)患者步态冻结(FOG)的疗效进行了研究。然而,研究结果仍存在争议。我们评估了新开发的振动触觉足部装置对帕金森病 FOG 患者冻结严重程度和步态测量的疗效:评估振动足部触觉装置对患有 FOG 的帕金森病患者的疗效:方法:对33名患有FOG的PD患者在 "停药 "状态下进行了检查。振动触觉足部装置的疗效通过步态协议进行评估,步态协议包括振动触觉刺激 "关闭 "和 "开启 "时的行走试验。两名运动障碍专家对行走试验进行了录像,以便进行离线评级。Opal 惯性传感器装置(128 Hz;Mobility Lab;APDM Inc.,美国俄勒冈州波特兰市)用于步态定量分析:结果表明,FOG 的发作次数减少了 33.1%(P 结论:振动触觉足部装置对 FOG 的治疗效果显著:振动触觉足部装置是一种有效的装置,可显著降低僵直的严重程度,并为经常僵直的帕金森病患者提供步态调节。该装置可在家庭环境中使用,以提高生活质量。
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引用次数: 0
Vascular Leukoencephalopathy Associated Chorea Due to A Heterozygous Htra 1 Variant: Novel Presentation of Cadasil Type II. Htra 1杂合子变异导致的血管性白质脑病相关性舞蹈症:卡达希尔 II 型的新表现。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-08-06 DOI: 10.1002/mdc3.14186
Alina Nazir, Ali Zafar, Edward Jones, Muhammad Awan
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引用次数: 0
Unilateral Upper-Limb Tremor Due to C6 Radiculopathy and Demyelinating Neuropathy. C6神经根病和脱髓鞘神经病导致的单侧上肢震颤。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-08-30 DOI: 10.1002/mdc3.14180
Sai A Nagaratnam, Nimalan Harinesan, Matthew Silsby, Victor S C Fung
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引用次数: 0
Long-Term Outcomes of GPi Deep Brain Stimulation in a Child with Glutaric Aciduria Type 1 (GA1). 1 型戊二酸尿症 (GA1) 患儿接受 GPi 脑深部刺激的长期疗效。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-08-12 DOI: 10.1002/mdc3.14185
Almudena Chacón, Olga Mateo-Sierra, Javier Ricardo Pérez-Sánchez, Beatriz De la Casa-Fages, Francisco Grandas, Pedro De Castro, Concepción Miranda
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引用次数: 0
Dystonia in a Patient with Genetically Proven Salih Ataxia Due to a Novel Truncating Variant: Expanding the Genotypic and Phenotypic Spectrum. 一名经基因证实的萨利共济失调患者因新型截短变异而出现肌张力障碍:扩大基因型和表型范围。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-06-27 DOI: 10.1002/mdc3.14152
Nitish Kamble, Vikram V Holla, Pavan Kumar Katragadda, Babylakshmi Muthusamy, Pramod Kumar Pal
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引用次数: 0
Prescription Drug Utilization among Patients with Essential Tremor: A Cross-Sectional Study of More Than 36,000 Patients. 本质性震颤患者的处方药使用情况:对 36,000 多名患者进行的横断面研究。
IF 2.6 4区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-01 Epub Date: 2024-07-07 DOI: 10.1002/mdc3.14155
Kandice A Kapinos, Elan D Louis

Background: Essential tremor (ET) is a chronic, progressive neurological disease that affects an estimated 7 million individuals in the United States (ie, 2.2% of the entire U.S. population). Despite its high prevalence, there are a few published studies on patterns of prescription medication use among patients.

Objective: The aim was to examine prescription drug medication use among ET patients.

Methods: This is a cross-sectional study of ET patients, age ≥40, with at least 1 prescription medication fill using the Optum's de-identified Clinformatics Data Mart Database from 2018 through 2019. We examined patterns of fills of key agents used to treat ET.

Results: The final sample comprised 36,839 ET patients in the United States; 89% had at least 1 prescription drug claim over a 2-year period, indicating that 9 of 10 ET patients take a medication to treat their disease. For each of the 3 most frequently prescribed medications, only a modest fraction (1/5 to 1/4) of patients were taking that medication. Adherence to these agents was 52% to 61%. A high percentage of patients had fills for more than 1 of the main agents we studied.

Conclusion: These data illustrate a need for medication in the ET population. There is only 1 FDA-approved medication to treat ET, propranolol, and less than 25% of ET patients used this drug during our study period. At the same time, no single agent was utilized by more than one quarter of ET patients, adherence was low, and use of multiple agents was common. For such a common disease, the pharmacotherapeutic landscape is impoverished.

背景:特发性震颤(ET)是一种慢性、进行性神经系统疾病,在美国约有 700 万人患病(占美国总人口的 2.2%)。尽管其发病率很高,但有关患者处方药使用模式的公开研究却寥寥无几:目的:研究 ET 患者使用处方药的情况:这是一项横断面研究,研究对象为年龄≥40 岁的 ET 患者,他们在 2018 年至 2019 年期间使用 Optum 的去标识化临床信息学数据集市数据库至少填充过一次处方药。我们研究了用于治疗 ET 的主要药物的配药模式:最终样本包括美国的 36839 名 ET 患者;89% 的患者在两年内至少报销过一次处方药,这表明每 10 名 ET 患者中就有 9 人服用药物治疗疾病。在 3 种最常用的处方药中,只有一小部分(1/5 至 1/4)患者在服用该药。这些药物的依从性为 52% 至 61%。在我们研究的主要药物中,有很高比例的患者需要服用一种以上的药物:这些数据说明了 ET 患者对药物的需求。美国食品及药物管理局只批准了一种治疗 ET 的药物--普萘洛尔,而在我们的研究期间,只有不到 25% 的 ET 患者使用过这种药物。同时,没有任何一种药物被超过四分之一的 ET 患者使用,用药依从性很低,使用多种药物的情况也很普遍。对于这样一种常见疾病,药物治疗的前景是贫乏的。
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Movement Disorders Clinical Practice
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