IMproving Symptomatic Treatment With Pyridostigmine and Amifampridine: a Randomized Double-blinded, Placebo Controlled Crossover Trial in Patients With Myasthenia Gravis (IMPACT-MG)

A randomized, double-blind, placebo controlled, crossover intervention study evaluating the effect of pyridostigmine (part 1) and amifampridine (part 2) in Myasthenia Gravis (MG).

开始日期2023-03-22

申办/合作机构

Academisch Ziekenhuis Leiden

[+1]

NCT05123053 / Recruiting临床2期IIT

Amifampridine for the Treatment of Transient Vocal Weakness After OnabotulinumtoxinA Injection for Spasmodic Dysphonia

Botox injections into the thyroarytenoid muscle are a predictable and effective treatment for SD, but typically result in transient symptoms of voice weakness and breathiness during the first 2-3 weeks after injection. Investigators hypothesize that voice weakness and breathiness after Botox treatment can be alleviated using amifampridine.

开始日期2021-10-28

申办/合作机构

Augusta University Research Institute

EUCTR2018-002405-64-IT / Not yet recruiting临床3期

Long term safety study of amifampridine phosphate in patients with Congenital Myasthenic Syndromes (CMS) - CMS-002

开始日期2019-05-06

申办/合作机构

Catalyst Pharmaceuticals, Inc.

100 项与磷酸阿米吡啶相关的临床结果

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100 项与磷酸阿米吡啶相关的转化医学

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100 项与磷酸阿米吡啶相关的专利（医药）

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686

项与磷酸阿米吡啶相关的文献（医药）

2024-04-01·Journal of affective disorders

Can an app increase health literacy and reduce the stigma associated with obsessive-compulsive disorder? A crossover randomized controlled trial

Article

作者: Del Valle, Gema ; Moritz, Steffen ; Arnáez, Sandra ; Chaves, Antonio ; Roncero, María ; García-Soriano, Gemma

BACKGROUND:

Obsessive-compulsive disorder (OCD) is a disabling condition with a high delay in seeking treatment. esTOCma is an app developed to increase mental health literacy (MHL) about OCD, reduce stigma, and increase the intention to seek professional treatment. It is a serious game and participants are asked to fight against the "OCD stigma monster" by accomplishing 10 missions. The aim of this study is to evaluate the effectiveness of this app in a community sample.

METHODS:

A randomized controlled trial with a crossover design was carried out. Participants were randomized to two groups: immediate use (iApp, n = 102) and delayed use (dApp, n = 106) of esTOCma. The iApp group started using the app at baseline until the game was over. The dApp group initiated at 10-days until the game finished. Participants were requested to complete a set of questionnaires at baseline and 10-day, 20-day and 3-month follow-ups.

RESULTS:

The Time×Group interaction effect was significant for the primary outcome measures: there was an increase in MHL and intention to seek help, and a decrease in stigma and OC symptoms, with large effect sizes, only after using the app. Changes were maintained (or increased) at follow-up.

LIMITATIONS:

The study did not include an active control group and some of the scales showed low internal consistency or a ceiling effect.

CONCLUSIONS:

This study provides first evidence for the effectiveness of esTOCma as a promising intervention to fight stigma and reduce the treatment gap in OCD.

TRIAL REGISTRATION:

ClinicalTrials.gov Identifier: NCT04777292. Registered February 23, 2021, https://clinicaltrials.gov/ct2/show/NCT04777292.

2024-04-01·Environmental research

The regulation effect of preventing soil nitrogen loss using microbial quorum sensing inhibitors

Article

作者: Zhuang, Guoqiang ; Gao, Jie ; Wang, Qiuying ; Wei, Jing ; An, Qiong ; Wan, Bin

Preventing soil nitrogen (N) losses driven by microbial nitrification and denitrification contributes to improving global environmental concerns caused by NO₃^--N leaching and N₂O emission. Quorum sensing (QS) signals regulate nitrification and denitrification of N-cycling bacteria in pure culture and water treatment systems, and mediate the composition of ammonia-oxidizing archaea (AOA) and ammonia-oxidizing bacteria (AOB) in activated sludge. However, whether disrupting QS could prevent soil N losses remains unclear. This study explored the feasibility of applying quorum sensing inhibitors (QSIs) as an innovative strategy to reduce N losses from agricultural soils. The two QSIs, penicillic acid and 4-iodo-N-[(3S)-tetrahydro-2-oxo-3-furanyl]-benzeneacetamide (4-iodo PHL), were more effective in reducing N losses than traditional inhibitors, including N-(n-butyl) thiophosphoric triamide and 3,4-dimethylpyrazole phosphate. After 36 days of aerobic incubation, penicillic acid and 4-iodo PHL inhibited nitrification by 39% and 68%, respectively. The inhibitory effects are attributed to the fact that 4-iodo PHL decreased the abundance of archaeal and bacterial amoA genes, as well as the relative abundance of Candidatus Nitrocosmicus (AOA), Candidatus Nitrososphaera (AOA), and Nitrospira (nitrite-oxidizing bacteria/comammox), while penicillic acid reduced archaeal amoA abundance and the relative abundance of Nitrosospira (AOB) and the microbes listed above. Penicillic acid also strongly inhibited denitrification (33%) and N₂O emissions (61%) at the peak of N₂O production (day 4 of anaerobic incubation) via decreasing nitrate reductase gene (narG) abundance and increasing N₂O reductase gene (nosZ) abundance, respectively. Furthermore, the environmental risks of QSIs to microbial community structure and network stability, CO₂ emissions, and soil animals were acceptable. Overall, QSIs have application potential in agriculture to reduce soil N losses and the associated effect on climate change. This study established a new method to mitigate N losses from the perspective of QS, and can serve as important basis of decreasing the environmental risks of agricultural non-point source pollution.

2024-02-01·The Journal of pharmacology and experimental therapeutics

Aminopyridines Restore Ventilation and Reverse Respiratory Acidosis at Late Stages of Botulism in Mice

Article

作者: Campbell, Charity J ; Jacobson, Alan R ; McNutt, Patrick M ; McClintic, William T ; Chandler, Zachary D ; Karchalla, Lalitha M ; Ondeck, Celinia A ; O'Brien, Sean W

Botulinum neurotoxin (BoNT) is a potent protein toxin that causes muscle paralysis and death by asphyxiation. Treatments for symptomatic botulism are intubation and supportive care until respiratory function recovers. Aminopyridines have recently emerged as potential treatments for botulism. The clinically approved drug 3,4-diaminopyridine (3,4-DAP) rapidly reverses toxic signs of botulism and has antidotal effects when continuously administered in rodent models of lethal botulism. Although the therapeutic effects of 3,4-DAP likely result from the reversal of diaphragm paralysis, the corresponding effects on respiratory physiology are not understood. Here, we combined unrestrained whole-body plethysmography (UWBP) with arterial blood gas measurements to study the effects of 3,4-DAP, and other aminopyridines, on ventilation and respiration at terminal stages of botulism in mice. Treatment with clinically relevant doses of 3,4-DAP restored ventilation in a dose-dependent manner, producing significant improvements in ventilatory parameters within 10 minutes. Concomitant with improved ventilation, 3,4-DAP treatment reversed botulism-induced respiratory acidosis, restoring blood levels of CO₂, pH, and lactate to normal physiologic levels. Having established that 3,4-DAP-mediated improvements in ventilation were directly correlated with improved respiration, we used UWBP to quantitatively evaluate nine additional aminopyridines in BoNT/A-intoxicated mice. Multiple aminopyridines were identified with comparable or enhanced therapeutic efficacies compared with 3,4-DAP, including aminopyridines that selectively improved tidal volume versus respiratory rate and vice versa. In addition to contributing to a growing body of evidence supporting the use of aminopyridines to treat clinical botulism, these data lay the groundwork for the development of aminopyridine derivatives with improved pharmacological properties. SIGNIFICANCE STATEMENT: There is a critical need for fast-acting treatments to reverse respiratory paralysis in patients with botulism. This study used unrestrained, whole-body plethysmography and arterial blood gas analysis to show that aminopyridines rapidly restore ventilation and respiration and reverse respiratory acidosis when administered to mice at terminal stages of botulism. In addition to supporting the use of aminopyridines as first-line treatments for botulism symptoms, these data are expected to contribute to the development of new aminopyridine derivatives with improved pharmacological properties.

133

项与磷酸阿米吡啶相关的新闻（医药）

2024-09-04

·药明康德

96%患者持续一年无复发！罗氏穿越血脑屏障小分子亮眼数据公布

▎药明康德内容团队编辑今日，罗氏（Roche）公布临床2期试验FENopta的开放标签扩展（OLE）研究48周新数据。结果表明，接受其口服布鲁顿酪氨酸激酶（BTK）抑制剂fenebrutinib治疗长达一年的复发性多发性硬化（RMS）患者中，高达96%患者没有发生疾病复发与进展。详细数据将公布于第40届欧洲多发性硬化治疗和研究委员会大会（ECTRIMS）当中。目前共有3项fenebrutinib相关的临床3期试验正在进行中，预计将于2025年底公布数据。多发性硬化症（MS）是一种慢性疾病，全球有超过280万患者。MS发生时，免疫系统异常攻击中枢神经系统（脑、脊髓和视神经）中神经细胞的髓鞘（对神经细胞的轴突起到绝缘和支持作用），导致炎症和损伤。这种损伤可能导致一系列症状，包括肌肉虚弱，疲劳和视力下降，最终可能导致残疾。大多数MS患者在20到40岁之间首次出现症状，使该疾病成为年轻成年人非创伤性残疾的主要原因。 FENopta是一项全球性的随机、双盲、安慰剂对照的2期研究，为期12周，旨在研究fenebrutinib对109名年龄在18-55岁之间RMS成年患者的疗效、安全性和药代动力学。完成FENopta研究的患者可以选择参加OLE研究。参与OLE研究的所有患者均接受fenebrutinib治疗，最长可达192周。其中有99例患者进入OLE，96例患者在一年后仍留在OLE。图片来源：123RF 之前公布为期12周的试验结果显示，fenebrutinib达到试验主要终点。该药物能够穿越血脑屏障，并且在大脑中的浓度达到可直接抑制炎症的水平。Fenebrutinib显著减少了活动性炎症标志物新发T1钆增强（T1 Gd+）颅脑病灶总数，以及代表疾病负荷或慢性病变负荷的新发或扩大的T2颅脑病灶总数。4周时观察到病灶快速缩小，在8周和12周时观察到T1 Gd+病灶相对缩小92%和90%，T2病灶相对缩小90%和95%。这次公布的OLE结果显示，在OLE期间，96%接受fenebrutinib治疗的患者在一年内没有复发，年复发率（ARR）为0.04，并且根据扩展残疾状况量表（EDSS）测量，患者48周内残疾程度没有发生变化。此外，根据磁共振成像（MRI）扫描结果，fenebrutinib持续抑制颅脑疾病活动。48周后，99%的患者不再出现T1 Gd+病灶。此外，与双盲期结束时相比，在48周OLE治疗期间持续接受fenebrutinib治疗的患者，其T2病灶的体积减少了三倍。 OLE期间中，fenebrutinib的安全性与之前报告的数据一致。发生在超过5%患者中最常见的不良事件（AE）包含尿路感染（8%）、COVID-19（7%）和咽炎（5%）。一例患者（1%）发生了严重不良事件。Fenebrutinib的安全性特征迄今为止已在超过2700人中受检视。 ▲Fenebrutinib简介（图片来源：罗氏官网） Fenebrutinib是一种在研的口服、可逆且非共价的BTK抑制剂，能够阻断BTK的功能。BTK是一种调控B细胞发育和活化的酶，同时也参与调控免疫系统中髓样细胞，如巨噬细胞和小胶质细胞的激活。临床前数据显示，fenebrutinib具有高效力和高选择性。Fenebrutinib显示出对BTK的选择性是对其他激酶的130倍，其高选择性和可逆性的特性可能降低分子的脱靶效应，从而有助于药品的长期安全性。罗氏目前正在进行三项3期临床试验，包括针对RMS的FENhance 1和2试验以及针对原发性进行性多发性硬化（PPMS）的FENtrepid试验。这些数据将评估fenebrutinib对多发性硬化谱系疾病进展的影响，预计将于2025年底公布。目前有多款MS在研疗法处于临床阶段。例如，今年4月，赛诺菲（Sanofi）公布其潜在“first-in-class”的CD40L靶向单抗frexalimab在2期试验中的积极结果，高达96%接受高剂量治疗的患者在48周治疗结束时没有发现新的脑部病灶。赛诺菲已启动相关3期临床试验对frexalimab作进一步检视。赛诺菲同时和Denali Therapeutics合作开发RIPK1小分子抑制剂SAR443820/DNL788用以治疗MS患者，该疗法目前处于2期临床阶段，预计在2025年完成试验。而由Zenas BioPharma开发的CD19和FcγRIIb靶向双功能单抗obexelimab目前也在2期临床阶段受检视。除了抗体与小分子类疗法，细胞与基因疗法也逐渐成为MS的研发方向之一。例如，Kyverna Therapeutics在今年4月宣布，在两名进展性多发性硬化患者的脑脊液中观察到其所开发的CD19靶向CAR-T疗法KYV-101细胞的存在和扩增，并且在一名患者中观察到鞘内抗体水平的下降，这意味着CAR-T细胞可以靶向中枢神经系统中的CD19阳性B细胞。而NeuroGenesis Bio公司的主打细胞疗法NG-01在2期试验中使得MS患者血清中神经丝轻链（NfL）水平一致降低，这是神经退行性病变和预测MS疾病进展的可靠生物标志物。百时美施贵宝（Bristol Myers Squibb）的CD19靶向CAR-T疗法CD19 NEX-T目前则在1期试验当中受检视其治疗MS患者的潜力。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] Roche’s fenebrutinib demonstrated near-complete suppression of disease activity and disability progression for up to 48 weeks in patients with relapsing multiple sclerosis. Retrieved September 4, 2024 from https://www.globenewswire.com/en/news-release/2024/09/04/2940215/0/en/Roche-s-fenebrutinib-demonstrated-near-complete-suppression-of-disease-activity-and-disability-progression-for-up-to-48-weeks-in-patients-with-relapsing-multiple-sclerosis.html[2] Late-breaking data for Roche’s BTK inhibitor fenebrutinib show brain penetration and significant reduction in lesions in patients with relapsing multiple sclerosis. Retrieved October 13, 2023, from https://www.roche.com/media/releases/med-cor-2023-10-13 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床结果临床2期临床3期

2024-08-26

·FierceBiotech

MBX files for IPO, seeking cash to take challenger to Ascendis' Yorvipath into phase 3

In addition to supporting its lead drug, MBX has earmarked some money to take an obesity candidate into the clinic. MBX Biosciences has added to the recent flurry of IPO filings. The biotech, which filed its paperwork weeks after raising $63.5 million privately, is seeking funding to take a would-be challenger to Ascendis Pharma’s rare endocrine disease drug Yorvipath into phase 3 development.Indiana-based MBX is built on technology designed to address the limitations of both unmodified and modified peptide therapies. By engineering peptides to improve their druglike properties, the biotech is trying to reduce the frequency of dosing, ensure consistent drug concentrations and otherwise establish product characteristics that improve clinical outcomes and simplify the management of diseases.MBX used the platform to create the hypoparathyroidism candidate MBX 2109. The biotech is trying to provide continuous exposure to parathyroid hormone (PTH) with once-weekly dosing. MBX 2109 was generally well tolerated in phase 1, with no serious drug-related effects, and is now in phase 2.Management is aiming to report top-line data in the third quarter of 2025 and advance the molecule into phase 3 using the IPO cash. The strategy puts the biotech on a collision course with Ascendis, a biotech that sells a once-daily PTH replacement therapy. MBX sees a need for a more convenient treatment that can normalize serum and urine calcium. AstraZeneca has a once-daily asset, eneboparatide, in phase 3. GLP-1, the peptide at the heart of the obesity drug boom, is central to the rest of MBX’s pipeline. The company has a once-weekly GLP-1 receptor antagonist, MBX 1416, in development. MBX sees the asset as a potential treatment of post-bariatric hypoglycemia, a chronic complication of weight loss surgery. The drug is in phase 1 testing. Data are due this year, and MBX plans to move into phase 2 using the IPO cash.MBX has also earmarked some money to take an obesity candidate into the clinic. The prospect, MBX 4291, is a GLP-1/GIP receptor co-agonist prodrug. Eli Lilly already sells a GLP-1/GIP receptor co-agonist, tirzepatide, as Mounjaro in diabetes and Zepbound in obesity. However, tirzepatide is given once a week. MBX is aiming to achieve once-monthly dosing when it files to test its asset in humans next year.Amgen’s bispecific GLP-1/GIP drug candidate AMG 133 could also support once-monthly dosing, but most molecules are targeting once-weekly administration. MBX is trailing Amgen, which is running a phase 2 trial of its once-monthly prospect.The biotech submitted its paperwork the day after Bicara Therapeutics and Zenas Biopharma filed to go public. Like MBX, Bicara and Zenas are seeking cash to take candidates into and through late-phase trials.

临床2期临床1期临床3期IPO

2024-08-24

·药事纵横

申请IPO！这家自身免疫生物技术公司手握一款III期双功能单抗

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 Zenas BioPharma周四申请首次公开募股（IPO），这标志着生物技术初创公司今年夏天的罕见举动。近几个月来，这家处于后期阶段的自身免疫生物技术公司仅次于其他几家药物开发商在纳斯达克的飞跃，其中包括专注于免疫学的公司Artiva Biotherapeutics和Alumis。中枢神经系统疾病生物技术公司Rapport Therapeutics也在6月初采取了这一举措。这家总部位于马萨诸塞州沃尔瑟姆的生物技术公司在披露由SR One牵头的2亿美元C轮融资几个月后，寻求以ZBIO的名义上市。该公司没有透露拟议IPO的规模。它设定了1亿美元的标准最高发行价，但这通常会在路演期间波动。 Zenas在CG Oncology和ArriVent Biopharma于1月下旬上市的同一周秘密提交了IPO申请，这或许表明它希望利用这些公司的势头，以及投资者和董事会成员在当月早些时候的摩根大通医疗保健会议上表现出的乐观情绪。这家初创公司满足了公共生物技术投资者在IPO长期枯竭期中青睐的一些条件：它处于临床阶段（第三阶段），并且处于该行业最热门的研发领域之一（自身免疫性疾病）。该公司的大部分工作都集中在obexelimab上，这是一种用于免疫球蛋白G4相关疾病的3期双功能单克隆抗体。它还启动了实验药物在多发性硬化症和系统性红斑狼疮中的2期测试，并处于温自身免疫性溶血性贫血的2/3期开放标签部分。 Zenas还与Viridian Therapeutics在甲状腺眼病方面建立了合作伙伴关系，与Dianthus Therapeutics在某些自身免疫性疾病方面建立了合作伙伴关系。根据 S-1 文件，今年前六个月，该公司在研发上花费了 5640 万美元，几乎与 2023 年全年的 6000 万美元花费相同。根据该文件，截至6月底，该公司拥有1.83亿美元的现金。持有该公司5%或以上股份的投资者包括Xencor、Enavate Sciences、SR One、Longitude Capital、Tellus BioVentures、Fairmount Funds Management、New Enterprise Associates、Norwest Venture Partners和Bristol Myers Squibb。具体的细分情况没有披露。 Xencor 最初推动了 obexelimab 的开发，但在 2021 年因在狼疮中期测试中失败而出售。领导这家生物技术公司的是创始人、董事长兼首席执行官朗尼·莫尔德（Lonnie Moulder），他曾共同创立并将Tesaro出售给葛兰素史克（GSK）。他也是投资者Tellus BioVentures的管理成员。 Moulder今年夏天早些时候告诉Endpoints News，Zenas希望将obexelimab推向美国和欧洲市场，同时在包括亚洲在内的其他地区进行许可。BMS在日本、韩国和澳大利亚等亚太国家拥有权利。

IPO临床3期临床2期

100 项与磷酸阿米吡啶相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10689	磷酸阿米吡啶	N07XX05	DB11640

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
Lambert-Eaton肌无力综合征	欧盟	SERB SA	2009-12-23
Lambert-Eaton肌无力综合征	冰岛	SERB SA	2009-12-23
Lambert-Eaton肌无力综合征	列支敦士登	SERB SA	2009-12-23
Lambert-Eaton肌无力综合征	挪威	SERB SA	2009-12-23

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
重症肌无力	临床3期	美国	Catalyst Pharmaceuticals, Inc.	2018-04-18
先天性肌无力综合征	临床3期	美国	Catalyst Pharmaceuticals, Inc.	2016-01-01
青少年脊髓性肌萎缩症	临床2期	意大利	Catalyst Pharmaceuticals, Inc.	2019-01-21
脊髓性肌萎缩	临床1期	-	Catalyst Pharmaceuticals, Inc.	-
脊髓延髓肌肉萎缩症	临床前	美国	Catalyst Pharmaceuticals, Inc.	-
多发性硬化症	药物发现	美国	BioMarin Pharmaceutical, Inc.	-

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03819660 (SMA3, CTgov)	临床2期	青少年脊髓性肌萎缩症	13	Amifampridine Phosphate 10 MG Oral Tablet	鏇繭餘鏇鬱廠襯鹽壓鑰(範憲齋廠顧窪鹽繭糧願) = 遞範壓襯鹹顧網鹹鏇廠鬱衊憲壓醖鬱鏇積鑰襯 (齋製獵鑰窪淵齋鬱積範, 淵遞願願遞鹽鹽鏇願繭 ~ 築簾壓膚淵蓋艱衊顧鹽) 更多	-	2023-11-30
NCT03304054 (CTgov)	临床3期	重症肌无力	93	Amifampridine Phosphate (Amifampridine Phosphate - MuSK)	憲醖蓋構鹽憲淵築鏇獵(衊齋範選鏇齋淵願築構) = 襯鬱選夢齋衊糧簾願壓鏇窪鬱淵鑰窪遞繭夢淵 (簾膚觸簾衊鹽築獵簾襯, 齋積蓋膚製繭遞鏇鏇鑰 ~ 鏇鏇鹽網簾簾膚鬱廠鑰) 更多	-	2021-08-13
				Placebo+amifampridine phosphate (Placebo - MuSK)	憲醖蓋構鹽憲淵築鏇獵(衊齋範選鏇齋淵願築構) = 選範繭壓鹽餘鬱願艱獵鏇窪鬱淵鑰窪遞繭夢淵 (簾膚觸簾衊鹽築獵簾襯, 鏇觸鬱構網壓顧積淵衊 ~ 觸糧顧獵積餘廠壓艱淵) 更多
NCT02090725 (3-4DAP, CTgov)	临床2期	Lambert-Eaton肌无力综合征 \| 肌肉无力	4	3-4 Diaminopyridine	積艱顧繭廠選艱廠廠淵(醖膚顧餘窪鹹鑰範壓淵) = 醖衊築鑰壓淵鏇膚網膚積膚簾餘鏇網餘醖鹹遞 (膚鑰鬱鏇襯鏇衊憲簾壓, 遞醖窪憲顧衊積窪廠衊 ~ 築鹽淵廠製鹽壓鑰襯窪) 更多	-	2019-05-21
NCT02970162 (Pubmed) 人工标引	临床3期	Lambert-Eaton肌无力综合征	26	Amifampridine	醖窪鹹顧襯鹽醖憲鏇齋(網夢積壓願膚壓齋鏇網) = Other measures of efficacy, including Clinical Global Impression-Improvement, 3TUG, and QMG limb domain score also improved. 鬱鹽遞膚顧選衊繭願艱 (鬱鑰獵醖選鏇選簾簾鏇 ) 更多	积极	2019-03-01
				Placebo
NCT02970162 (CTgov)	临床3期	Lambert-Eaton肌无力综合征	26	Amifampridine Phosphate (Amifampridine Phosphate)	遞網遞餘糧構選鹹襯簾(艱壓糧製襯網鏇鑰鹽襯) = 糧鹹鏇願餘觸窪鑰網獵壓醖衊遞鏇鬱鑰艱鏇範 (願壓製憲鬱窪選衊觸廠, 鑰襯糧蓋夢鬱製膚廠網 ~ 壓廠餘鬱蓋窪鑰夢簾壓) 更多	-	2018-12-24
				Placebo Oral Tablet (Placebo (for Amifampridine Phosphate))	遞網遞餘糧構選鹹襯簾(艱壓糧製襯網鏇鑰鹽襯) = 觸蓋蓋糧衊範夢構觸艱壓醖衊遞鏇鬱鑰艱鏇範 (願壓製憲鬱窪選衊觸廠, 積鹹願簾顧鬱廠鏇簾鬱 ~ 製獵願獵願鑰鬱顧衊獵) 更多
P6.452 (AAN2018)	N/A	先天性肌无力综合征	2	3,4 DAP	壓廠繭遞廠糧網鹽構鬱(積積鏇積製製獵構齋網) = 鑰網淵構積鏇網醖網簾窪醖壓簾築蓋醖願顧鑰 (製鑰顧築鏇蓋廠襯範淵 )	积极	2018-04-10
NCT01377922 (CTgov)	临床3期	Lambert-Eaton肌无力综合征	38	Placebo (Placebo)	窪選醖蓋鏇鏇簾鬱獵窪(壓蓋蓋選膚廠積醖遞願) = 構夢鏇構鹹醖醖壓鹽壓醖衊膚鹽醖繭簾觸糧選 (積蓋餘齋膚艱憲窪願願, 鏇窪構繭艱築淵蓋觸選 ~ 製願鹹鏇獵鹽餘廠艱簾) 更多	-	2018-01-04
				Amifampridine Phosphate (Amifampridine Phosphate)	窪選醖蓋鏇鏇簾鬱獵窪(壓蓋蓋選膚廠積醖遞願) = 願憲廠夢遞選鹹艱鏇襯醖衊膚鹽醖繭簾觸糧選 (積蓋餘齋膚艱憲窪願願, 憲餘鏇艱積淵艱鹹觸願 ~ 鏇鹹糧窪窪鏇鏇壓觸願) 更多