An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed the MEX-DM-302 Study (ATLAS Study)

This is an open-label extension study intended to evaluate the long-term safety and efficacy of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2) who have completed the parent study MEX-DM-302.

开始日期2025-02-27

申办/合作机构

Lupin Ltd

[+1]

NCT06862596

/ Recruiting临床2/3期IIT

A Multicenter, Randomized, Placebo-controlled, Double-blind Clinical Trial: the Efficacy and Safety of Mexiletine Hydrochloride for Amelioration of Motor Dysfunction in Spinal and Bulbar Muscular Atrophy

The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy.
The main questions it aims to answer are:
Does mexiletine hydrochloride improve the ALSFRS-R score in spinal and bulbar muscular atrophy patients?
Participants will:
Take mexiletine hydrochloride or a placebo every day for 3 months Visit the hospital once every 4 weeks for evaluations.

开始日期2025-02-26

申办/合作机构

Nagoya University

CTR20243596

/ CompletedN/A

南京泽恒医药技术开发有限公司研制的盐酸美西律片与KYOWA CritiCare Co., Ltd公司持证的盐酸美西律片（商品名：Mexiletine Hydrochloride ®，规格：50 mg）在中国健康受试者中进行的随机、开放、单次给药、两周期、两序列、交叉、空腹及餐后状态下的生物等效性研究

以南京泽恒医药技术开发有限公司研制的盐酸美西律片（规格：50 mg）为受试制剂，以持证商为KYOWA CritiCare Co., Ltd公司的盐酸美西律片（商品名：Mexiletine Hydrochloride®，规格：50 mg）为参比制剂，考察两制剂在空腹及餐后状态下单次给药的药代动力学参数及相对生物利用度，评价两制剂是否具有生物等效性。同时评价两种制剂在健康人体中的安全性和耐受性。

开始日期2024-10-24

申办/合作机构

南京泽恒医药技术开发有限公司

100 项与盐酸美西律相关的临床结果

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100 项与盐酸美西律相关的转化医学

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100 项与盐酸美西律相关的专利（医药）

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3,010

项与盐酸美西律相关的文献（医药）

2025-03-01·Journal of clinical neuromuscular disease

What is in the Myopathy Literature?

Review

作者: Isfort, Michael ; Lacomis, David

Abstract:

This update begins with the incidence and features of statin-associated muscle symptoms, which may often be misattributed. Articles on potential muscle mitochondria dysfunction from statins follow, along with recommendations for possibly avoiding statins in some patients with genetic myopathies. Next, autoimmune myopathies, including immune-mediated necrotizing myopathy, myositis with antimitochondrial antibodies, and overlap myositis with lupus, as well as the role of myxovirus protein A identification in muscle specimens, are addressed. The next section includes reports on the significance of elevated serum aldolase with normal creatine kinase and recommended approaches to evaluate a patient with rhabdomyolysis. A cluster of reports on muscle imaging, particularly using ultrasound and magnetic resonance imaging, are covered. They include studies of inherited and inflammatory myopathies and neck extensor myopathy on topics such as imaging features, patterns of involvement, diagnostic utility, and correlation with histopathology. Last, there are discussions on mexiletine versus lamotrigine for nondystrophic myotonias and the treatment of fatty acid oxidation disorders in adults.

2025-03-01·KOREAN JOURNAL OF PHYSIOLOGY & PHARMACOLOGY

Human induced pluripotent stem cell-cardiomyocytes for cardiotoxicity assessment: a comparative study of arrhythmiainducing drugs with multi-electrode array analysis

Article

作者: Park, Soon-Jung ; Park, Yun-Gwi ; Park, Na Kyeong ; Moon, Sung-Hwan ; Choi, Seong Woo ; Choi, Ji-Hee ; Choi, Hyung Kyu

Reliable preclinical models for assessing drug-induced cardiotoxicity are essential to reduce the high rate of drug withdrawals during development. Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) have emerged as a promising platform for such assessments due to their expression of cardiacspecific ion channels and electrophysiological properties. In this study, we investigated the effects of eight arrhythmogenic drugs-E4031, nifedipine, mexiletine, JNJ303, flecainide, moxifloxacin, quinidine, and ranolazine-on hiPSC-CMs derived from both healthy individuals and a long QT syndrome (LQTS) patient using multielectrode array systems. The results demonstrated dose-dependent changes in field potential duration and arrhythmogenic risk, with LQTS-derived hiPSC-CMs showing increased sensitivity to hERG channel blockers such as E4031. Furthermore, the study highlights the potential of hiPSC-CMs to model disease-specific cardiac responses, providing insights into genetic predispositions and personalized drug responses. Despite challenges related to the immaturity of hiPSC-CMs, their ability to recapitulate human cardiac electrophysiology makes them a valuable tool for preclinical cardiotoxicity assessments. This study underscores the utility of integrating patientderived hiPSC-CMs with advanced analytical platforms, such as multi-electrode array systems, to evaluate drug-induced electrophysiological changes. These findings reinforce the role of hiPSC-CMs in drug development, facilitating safer and more efficient screening methods while supporting precision medicine applications.

2025-02-16·ChemSusChem

Stable and Versatile Pd Precursors for the Preparation of Robust Pd Catalysts Under Continuous‐Flow

Article

作者: Abe, Yuka ; Yokoyama, Takashi ; Sakurada, Naoya ; Yamada, Tsuyoshi ; Kobayashi, Kwihwan ; Ikawa, Takashi ; Niwa, Kosuke ; Sajiki, Hironao

Abstract:

To achieve global sustainability, the chemical and engineering communities require the development of versatile precursors that can be used to synthesize robust catalysts. To meet this demand, we developed a new Pd precursor for highly efficiently incorporating fine Pd‐metal into supports. An atmospherically stable Pd precursor (Pd‐80) was prepared by thermally promoting the aerobic oxidation of tetrakis(triphenylphosphine)palladium. The physical properties of Pd‐80 were investigated by NMR spectroscopy, SEM, XPS, solvent‐relaxation NMR spectroscopy, and dynamic light scattering (DLS) experiments. We also prepared a cordierite‐supported Pd catalyst (Pd/cordierite) by stirring Pd‐80 and cordierite powder in chloroform at room temperature. Pd/cordierite selectively catalyzed the hydrogenation of various reducible functional groups, including alkynes, azides, nitro groups, olefins, CO2Bn, N‐Cbz, O‐Bn, aromatic ketones, and styrene oxide, in continuous‐flow hydrogenation reactions. The Pd/cordierite‐catalyzed continuous‐flow hydrogenation of nitrobenzene derivatives afforded the corresponding anilines, with catalytic activities maintained for over 250 h of continuous operation with a turnover number (TON) of 61,090.

项与盐酸美西律相关的新闻（医药）

2025-02-17

·BioSpace

Opinion: Companies Take Multiple Tacks Against Myotonic Dystrophy Type 1

horillaz/ Getty Images With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this disease space alongside Avidity Biosciences, Lupin and AMO Pharma. Last month, Dyne Therapeutics announced positive results in a Phase I/II trial of its candidate DYNE-101. The milestone places Dyne among the companies closest to developing a disease-modifying treatment for myotonic dystrophy type 1. Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy caused by DMPK mutations on chromosome 19. It affects muscles and many organs across multiple systems that depend on muscular activity. Also known as Steinert disease, it is characterized by the inability to relax muscles at will. DM1 affects 140,000 people in the United States alone, and although there are drugs that address its symptoms, there are no currently approved disease-modifying treatments for the condition. There are, however, more than two dozen investigational drugs in development for DM1. Among the companies looking to fill the unmet need is Dyne, whose DYNE-101 is an antisense oligonucleotide fused with a fragment antibody that binds to the transferrin receptor 1 in muscles. In the Phase I/II ACHIEVE trial , key findings included DMPK RNA level reductions, CASI-22 improvements, early and sustained improvement in myotonia as measured by grip myotonia assessment, and multiple other strength and timed assessments. The trial utilized a multiple ascending dose cohort to identify 6.8 mg/kg every 8 weeks as the optimal dose for future studies. Dyne stated in its press release that it plans to initiate a global registrational expansion cohort of the trial to potentially support a submission for FDA Accelerated Approval in the first half of 2026. Meanwhile, Avidity Biosciences is developing AOC 1001, an antibody oligonucleotide conjugate that targets the DMPK gene to treat DM1. Also known as del-desiran, this investigational treatment is currently being studied in the Phase III HARBOR trial and Avidity has announced plans to submit marketing applications in 2026. In March 2024, Avidity announced positive long-term del-desiran data from the Phase II MARINA-OLE trial showing reversal of disease progression in people living with DM1 across multiple endpoints compared to a matched natural history study population over one year. Those endpoints included grip myotonia assessment, muscle strength and activities of daily living. According to Avidity, additional data from the ongoing Phase I/II MARINA trial for AOC 1001, in which patients were split into placebo-controlled single-dose and multiple ascending dose arms, will be released in the first quarter of this year. While clinical-stage investigational oligonucleotide-based therapy candidates are catching up, repurposed drugs such as metformin, tideglusib and mexiletine are also undergoing Phase III trials to treat DM1. Metformin , a biguanide currently approved for type 2 diabetes mellitus, is currently slated for Phase III trials in DM1. In previous Phase II trials, metformin increased the 6-minute walking distance for DM1 patients versus placebo. While it has positive benefits ranging from anticancer effects to cardiovascular protection to neuroprotection, metformin is also associated with adverse effects such as gastrointestinal discomfort and lactic acidosis in high doses. Another approach is AMO Pharma ’s tideglusib (also known as AMO-02), a glycogen synthase kinase 3 beta inhibitor currently in Phase III clinical trials. Despite not reaching its primary endpoint in the Phase II/III REACH-CDM trial due to a strong placebo effect, tideglusib had achieved clinically and statistically significant benefits in various functional and objective assessments as of May 2024. As a result, after meeting with the FDA, AMO Pharma is forging ahead with a Phase III trial of tideglusib. Elsewhere, Lupin is working on repurposing mexiletine, a class I antiarrhythmic drug that is currently in Phase III trials for DM1. In contrast to previous trials that used mexiletine capsules for immediate-release effects, Lupin intends to use mexiletine granules for prolonged-release oral suspension formulations for its Phase III HERCULES and ATLAS trials. Like tideglusib, mexiletine reached Phase III despite less-than-stellar mid-stage results. In its case, Phase II trial data demonstrated no significant change in 6-minute walk distance at 6 months for patients on mexiletine capsules compared to placebo. Also worthy of mention is ARTHex. While clinical data is scant for the company’s ATX-01, it was enrolling patients into its Phase I/IIa ArthemiR trial as of October 2024. The drug is based on microRNA technology. As progress currently stands, antibody-oligonucleotide conjugates appear to be more effective at targeting the genetic source of DM1 and have a more convincing mechanism of action than small molecules and repurposed drugs. However, even though genetic therapies are advancing quickly, the DM1 space is risky. Consider that Ionis Pharmaceuticals stopped working on its antisense compound IONIS-DMPK-2.5Rx in January 2017 after failing to achieve adequate concentration levels in muscles in Phase I/II trials. If all goes well in the late-stage gene therapy space for DM1, expect new treatments to hit the market within the next three to four years at the earliest.

临床3期寡核苷酸临床2期临床结果上市批准

2025-02-05

·CPHI制药在线

20年来首款新机制镇痛药重磅来袭

关注并星标CPHI制药在线 2025年1月30日，Vertex Pharmaceuticals宣布FDA批准Suzetrigine上市，用于治疗中至重度急性疼痛，这也是20多年来首款非阿片类镇痛药。 Suzetrigine(VX-548)是 NaV1.8 通道的靶向抑制剂。NaV1.8 是一种电压门控钠通道，选择性地表达于外周痛觉神经元，传递疼痛信号。Suzetrigine 通过高选择性抑制 NaV1.8通道，能有效缓解疼痛。本次获 FDA 批准，Suzetrigine 的疗效验证主要基于 2 项针对急性手术疼痛的随机、双盲、安慰剂和活性的对照试验。一项试验是在腹部整形术后，另一项是在拇囊切除术后。 2 项试验的都表明，与安慰剂相比，高剂量（初始剂量 100mg，之后以 50mg/12h 维持治疗）的Suzetrigine 可在 48 小时内有效减轻疼痛；与安慰剂相比，两组 SPID48（48 小时内疼痛强度差异的时间加权总和评分）的平均差异分别为 48.4 和 29.3。新机制镇痛靶点：Nav1.8 疼痛作为心脑血管疾病和肿瘤之后的第三大健康挑战，驱动了止痛药市场的快速增长，该市场扩张速度紧跟肿瘤药物之后，反映出临床中对更有效镇痛方案的迫切需求。据Mordor intelligence调查显示，2021年全球疼痛管理市场价值794亿美元，预计到2027年将增长至1207亿美元，复合年增长率为7.39%，因此镇痛药市场被认为是继GLP-1减肥药之后的下一个千亿金矿。鉴于阿片类药物滥用与成瘾问题严峻，医药界正集中力量探索新型非阿片类、无成瘾性的高效镇痛药物。疼痛信号传递机制与细胞膜上的电压控制钠离子通道（Nav）密切相关，尤其是通过调控Nav通道活性可以干预疼痛过程。Nav通道家族包含九个亚型（Nav1.1至Nav1.9），这些亚型分布广泛，其中Nav1.8和Nav1.9主要存在于外周神经系统。尽管抑制这些通道活动是疼痛治疗的一个方向，但由于Nav亚型的多样性及其在心脏、中枢神经系统等关键部位的存在，导致现有非选择性Nav抑制剂（例如拉莫三嗪、拉科酰胺、美西律）可能产生广泛的副作用，限制了它们的临床应用和安全性。 Nav1.7作为镇痛研究的重点之一，因其在人类遗传学研究中的良好验证而受到广泛关注。研究表明，Nav1.7的突变与遗传性疼痛综合症密切相关，这使得其成为了潜在的治疗靶点。然而，多个Nav1.7抑制剂化合物在临床试验中的表现不尽如人意，主要包括未能有效作用于脊柱分布、靶点占位不足、生物利用度差等问题。这些因素导致了Nav1.7抑制剂在实际应用中的局限性。此外，临床实验设计和靶点选择性也影响了开发结果，使得研究者们面临着重重挑战。这使得Nav1.7作为镇痛药物开发的靶点逐渐被取代，研究者们将注意力转向了Nav1.8这一亚型。 Nav1.8在痛觉信号传导中发挥着关键作用，主要表达于伤害感受神经元，并且属于对河豚毒素不敏感的钠离子通道。与Nav1.7不同的是，Nav1.8不参与中枢神经系统的活动，其抑制剂能够在提供有效镇痛效果的同时，避免成瘾性和对运动功能的不良影响。因此，Nav1.8成为了具有高选择性的疼痛治疗靶点。研究显示，通过靶向Nav1.8，可以有效减轻慢性疼痛而不引发依赖或其他副作用，这为新一代镇痛药物的开发提供了新的方向。 Vertex开了一个好头 Vertex曾经陆续将4款NaV1.8抑制剂(VX-128、VX-961、VX-150、VX-584)推进到临床试验阶段，其中VX-128和VX-961皆因I期临床试验PK和安全性问题分别于2018年和2020年终止。VX-150虽然PK和暴露量有所改善且也通过临床概念验证试验，但因2021年VX-584在I期临床试验中具有更明显安全性、PK和暴露量优势，因此VX-150亦遭放弃，Verte进而继续开发VX-548并最终取得临床试验成功。在行业媒体Evaluate发布的盘点文章中，VX-548疗法被列为潜在重磅疗法。有Leerink Partners分析师表示，当前的疼痛市场与两年前的肥胖市场有着相似之处，他预计到2030年VX-548的年销售额可能会达到51亿美元。然而，真正的临床需求并不是另一种急性疼痛药物，因为患者已经有很多现有且便宜的药物得到了有效且安全的的治疗（例如阿片类药物）但是对于慢性疼痛而言，非阿片类的镇痛药物意义就很重大了。VX-548有可能成为一种新的有效的慢性疼痛治疗方法，这是患者需求最大的领域，VX-548同时布局了性糖尿病神经病变（DPN）和慢性腰骶神经根病（LSR)。 DPN方面，尽管去年12月宣布了积极结果，但该试验的样本量不足以显示与临床对照药物普瑞巴林的疗效对比，并且没有安慰剂组。因此，Vertex宣布他们计划在2024年下半年启动一个更全面的III期项目。 LSR方面，在2期研究中VX-548虽然达到主要终点——即显著减轻了疼痛。然而，安慰剂组同样显示出明显的疼痛减轻（约 2 分），治疗组与安慰剂组之间的差异并不显著，仍需推向III20期临床进一步确定疗效。 Vertex钠离子通道抑制剂管线图片来源: Vertex官网总体而言，VX-548此次虽然顺利获批急性疼痛适应症，但在更具商业潜力的慢性疼痛适应症的开发上仍任重道远。在Vertex的镇痛管线中，除了VX-548在急性镇痛和慢性镇痛上的布局外，还正在开发第三代NaV1.8抑制剂VX-993和VX-973，旨在提供更安全、有效的非阿片类镇痛方案。这两种化合物已进入I期临床阶段，正在健康受试者中评估其安全性、耐受性和药代动力学特性。VX-993和VX-973有望优化药效以实现良好的镇痛效果。据丁香园 Insight 数据库显示，全球共有 30 款靶向 NaV1.8 的在研新药（仅统计活跃状态），有 12 款在研 NaV1.8 抑制剂已进入针对疼痛适应症的临床阶段。在国内，目前也有多款 NaV1.8 抑制剂已经进入临床阶段，包括恒瑞（Ⅱ 期）、济民可信（Ⅱ 期）、健康元（Ⅰ 期）、海博为（Ⅰ 期）。图片来源:贝壳社 JMKX000623是上海济煜自主研发的高选择性NaV 1.8）抑制剂,其针对糖尿病周围神经痛的Ⅱ 期临床正在进行中。早在2022年JMKX000623就实现出海，芬兰药企ORION以1500万欧元首付款买下了这款药物的海外权益,足以体现此产品的不俗价值。巧合的是，海博为药业的HBW-004285片也在近期完成了Ⅰ期临床。从已获得的临床研究数据显示，相比全球首个刚刚获批上市的Nav1.8抑制剂Journavx（即VX-548），HBW-004285片安全性更高（无任何一级以上不良反应）、起效速度更快，药代动力学更优，且无性别差异，有望超越VX-548；在临床前头对头对比实验中，HBW-004285无论是生物活性还是药代动力学性质，都显著优于Vertex下一代Nav 1.8 抑制剂 VX-993，是具有成为Best In Class潜力的药物分子。参考来源： 1. 各企业官网 2. CDE官网扫码领取CPHI & PMEC China 2025展会门票【智药研习社近期直播】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

上市批准

2024-11-27

·PRNewswire

Myotonic Dystrophy Clinical Trial Pipeline Boom as Over 20 Companies Leading the Charge in Research and Development | DelveInsight

An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion. LAS VEGAS, Nov. 27, 2024 /PRNewswire/ -- DelveInsight's ' Myotonic Dystrophy Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain. Key Takeaways from the Myotonic Dystrophy Pipeline Report DelveInsight's myotonic dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for myotonic dystrophy treatment. Key myotonic dystrophy companies such as Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others are evaluating new myotonic dystrophy drugs to improve the treatment landscape. Promising myotonic dystrophy pipeline therapies such as AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others are under different phases of myotonic dystrophy clinical trials. On November 7, 2024, ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced that the US Food and Drug Administration has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1. In November 2024, Dyne Therapeutics announced that the US Food and Drug Administration has cleared the Investigational New Drug application for DYNE-101, which is being evaluated in the ongoing, global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose). In August 2024, PepGen announced that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company's clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance for the treatment of myotonic dystrophy. In May 2024, Avidity Biosciences announced that the US Food and Drug Administration granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1. In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14th International Myotonic Dystrophy Consortium (IDMC-14) Meeting titled "FREEDOM-DM1: Phase 1 Study Design to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1." In March 2024, Entrada achieved a milestone under its global collaboration with Vertex related to the clinical advancement of Vertex's Phase I/II clinical trial of VX-670, which triggered a USD 75 million payment. The Company expects to receive this payment in the second quarter of 2024. The Company is eligible to receive up to USD 485 million, inclusive of milestones achieved to date, for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement. In February 2024, PepGen Inc. announced that the US Food and Drug Administration had granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1. In January 2024, Juvena Therapeutics receives the FDA Orphan Drug Designation for JUV-161 for the treatment of Myotonic Dystrophy Type 1. Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape. Myotonic Dystrophy Overview Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene. Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia. Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks. Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report: Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials Myotonic Dystrophy Therapeutics Assessment The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Myotonic Dystrophy Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Myotonin protein kinase expression inhibitors, RNA interference, Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, MicroRNA inhibitor, RNA inhibitors, RNA splicing modulators Key Myotonic Dystrophy Companies: Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others. Key Myotonic Dystrophy Pipeline Therapies: AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others. Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs Table of Contents For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs Related Reports Myotonic Dystrophy Market Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myotonic dystrophy companies, including Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, among others. Myotonic Dystrophy Epidemiology Forecast Myotonic Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

快速通道临床2期孤儿药

100 项与盐酸美西律相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D00639	盐酸美西律	C01BB02	DB00379

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
非营养不良性肌强直综合征	欧盟	Lupin Ltd	2018-12-18
非营养不良性肌强直综合征	冰岛	Lupin Ltd	2018-12-18
非营养不良性肌强直综合征	列支敦士登	Lupin Ltd	2018-12-18
非营养不良性肌强直综合征	挪威	Lupin Ltd	2018-12-18
肺脓肿	日本	Chemix, Inc.	2005-07-08
腹膜炎	日本	Chemix, Inc.	2005-07-08
肺炎	日本	Chemix, Inc.	2005-07-08
心律失常	日本	Taiyo Pharma Co., Ltd.	1985-01-31
糖尿病性神经病变	日本	Taiyo Pharma Co., Ltd.	1985-01-31

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
肌强直性营养不良	临床3期	法国	Lupin Ltd	2021-09-03
肌强直性营养不良	临床3期	-	Lupin Ltd	2021-09-03
HIV感染	临床2期	美国	Boehringer Ingelheim GmbH	2001-08-31
HIV感染	临床2期	坦桑尼亚	Boehringer Ingelheim GmbH	2001-08-31
痛性神经病变	临床2期	美国	Boehringer Ingelheim GmbH	2001-08-31
痛性神经病变	临床2期	坦桑尼亚	Boehringer Ingelheim GmbH	2001-08-31

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临床结果

适应症

分期

评价

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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


ESC2024	N/A	Timothy综合征	-	Mexiletine	簾簾選遞製顧鹹夢齋獵(繭築襯醖範構醖鹹鏇積) = 糧廠繭壓窪願憲選淵鑰選襯夢齋廠築觸願齋艱 (廠願醖蓋鏇膚壓糧衊願 )	-	2024-09-01
AAN2024	N/A	偏头痛	-	Mexiletine 450 mg	鹹獵願鹽觸遞淵築廠構(餘壓鬱願糧夢醖鏇齋膚) = Dizziness during lidocaine administration (p-value=0.0008) and lower tolerated lidocaine infusion rates (p-value=0.001) were predictive of shorter number of days on mexiletine treatment. A lower starting dose should be considered on these patients 觸艱積築醖淵範鑰網鬱 (遞簾膚獵鏇鏇淵鹹齋壓 ) 更多	-	2024-04-09
				Mexiletine 600 mg
MDS2023	N/A	肌强直	183	Mexiletine	獵築醖餘襯簾糧襯繭鏇(鑰衊醖餘鹽艱鏇範鹽獵) = mexiletine significantly causes some GIT symptoms compared to placebo (RR 3.7, 95% CI [1.79 to 7.64]) 觸壓醖窪醖窪艱範淵糧 (壓製鹽窪艱獵網積簾夢 )	积极	2023-08-27
EAN2023	N/A	-	-	Mexiletine	鬱繭築繭蓋鹽願醖醖艱(衊遞顧蓋選遞鹽鏇衊醖) = 膚顧觸觸網遞繭蓋積範艱顧壓鑰齋簾顧選鏇網 (鏇憲鹽願鹹獵鬱蓋選顧 )	-	2023-06-28
ESC2020	N/A	QT间期延长综合征2	20	mexiletine	醖壓衊鑰窪簾顧淵艱廠(觸夢衊衊廠積觸齋網鏇) = 顧鑰齋蓋觸網壓鑰顧繭積願餘窪餘齋顧淵鏇窪 (選範鹽簾淵獵鏇襯鑰簾 )	-	2020-08-28
2531 (AAN2020)	临床2期	肌萎缩侧索硬化	20	Placebo	繭廠蓋顧醖顧餘顧憲糧(顧築積衊壓窪醖鹹範鏇) = 壓醖鑰餘淵膚網願艱蓋獵憲簾繭醖網鹽艱獵蓋 (淵淵製鬱蓋膚獵獵鹹製, 2.2) 更多	积极	2020-04-14
				Mexiletine 300 mg	積淵壓願廠襯鑰鏇齋廠(壓憲醖簾鏇製鏇夢築簾) = 築蓋淵襯餘積簾蓋醖簾積築窪壓鹹膚願壓積構 (鬱範製網鹽衊醖遞繭選, 0.315 ~ 1.102)
NCT02781454 (Mexiletine-2, CTgov)	临床2期	肌萎缩侧索硬化	20	Mexiletine (Mexiletine, 300 Milligrams)	鹹壓蓋顧網鬱鏇積鑰鹽(鑰壓蓋繭遞廠簾艱醖築) = 顧鬱遞壓餘願齋餘網憲願憲憲構淵網餘築艱網 (獵積廠繭壓繭窪壓餘製, 2.323) 更多	-	2019-12-05
				Mexiletine (Mexiletine, 600 Milligrams)	鹹壓蓋顧網鬱鏇積鑰鹽(鑰壓蓋繭遞廠簾艱醖築) = 鏇襯壓範顧壓獵簾觸廠願憲憲構淵網餘築艱網 (獵積廠繭壓繭窪壓餘製, 2.207) 更多
WCN2019	N/A	非营养不良性肌强直综合征	-	Safinamide	構顧構範鑰襯鏇顧衊憲(窪蓋窪糧艱觸繭糧選衊) = 憲蓋鏇遞醖齋製艱鏇餘鬱範簾憲獵選夢夢襯憲 (獵繭醖蓋鬱鏇餘艱窪鬱 ) 更多	-	2019-10-15
				Mexiletine	構顧構範鑰襯鏇顧衊憲(窪蓋窪糧艱觸繭糧選衊) = 齋獵願鹽網襯鏇淵衊醖鬱範簾憲獵選夢夢襯憲 (獵繭醖蓋鬱鏇餘艱窪鬱 ) 更多
NCT02260388 (PAIN-CONTRoLS, CTgov)	临床4期	疼痛 \| 感觉性多发性神经病	402	Nortriptyline (Nortriptyline)	窪淵窪獵齋醖衊網獵築(壓製襯築壓獵製繭窪觸) = 築鏇觸鬱夢顧襯簾顧淵網願鹹淵積顧構網齋範 (鏇鹽鏇膚淵範築齋鹹遞, 8.9) 更多	-	2018-07-06
				Duloxetine (Duloxetine)	窪淵窪獵齋醖衊網獵築(壓製襯築壓獵製繭窪觸) = 蓋繭鏇糧鹽積觸觸顧艱網願鹹淵積顧構網齋範 (鏇鹽鏇膚淵範築齋鹹遞, 9.9) 更多
AAN2018	N/A	非营养不良性肌强直综合征	30	Mexiletine	鹽淵醖夢艱選艱範構構(壓醖淵鏇選蓋顧鬱糧壓) = 鬱窪願繭醖製蓋構膚淵獵選鑰衊製鏇窪衊壓襯 (獵繭醖鹹糧餘簾製觸積 ) 更多	积极	2018-04-10