罕见病知识库 RareSeen

多灶性运动神经病

Multifocal motor neuropathy

定义 英文原文(暂无中文)

Multifocal motor neuropathy (MMN) is a rare acquired immune-mediatedneuropathy characterized clinically by a purely motor deficit with conduction block and asymmetric multifocal weakness, fasciculations, and cramping.

别名

多灶性运动神经病伴传导阻滞

基本事实

遗传方式
未知
发病年龄
成年期
患病率
1-9 / 100 000

临床表型 12

极常见 99–80%1

  • 进行性远端肌无力 HP:0009063

常见 79–30%11

  • 神经节苷脂代谢异常 HP:0004345
  • 肌束震颤 HP:0002380
  • 功能性运动障碍 HP:0004302
  • 脑脊液蛋白浓度增加 HP:0002922
  • 四肢肌肉无力 HP:0003690
  • 伸腕受限 HP:0006251
  • 运动传导阻滞 HP:0012078
  • 肌肉痉挛 HP:0003394
  • 进行性肌无力 HP:0003323
  • 腱反射减低 HP:0001315
  • 第三指伸肌无力 HP:0009077

近两年的全球研究 279L2

2024/10 起在 Europe PMC 检索所得,按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务,尚未引入。

  • 2026-09
    The role of leukocyte p38 MAP kinase in immune-mediated peripheral polyneuropathies: distinct modulation by intravenous immunoglobulins
    Journal of neurology · DOI · Europe PMC
  • 2026-09综述开放获取
    Neurovascular Unit Dysfunction and Neural Barrier Pathology in Dysautonomia: Molecular Links to Cardiovascular Autonomic Failure
    International journal of molecular sciences · DOI · Europe PMC
  • 2026-09系统综述开放获取
    Comparative efficacy, safety, and functional recovery of neonatal fc receptor inhibitors and conventional immunotherapies for chronic inflammatory demyelinating polyradiculoneuropathy: a systematic review and network meta-analysis
    Frontiers in immunology · DOI · Europe PMC
  • 2026-09
    Guidelines on the Use of Therapeutic Apheresis in Clinical Practice-Evidence-Based Approach From the Writing Committee of the American Society for Apheresis: The Tenth Special Issue
    Journal of clinical apheresis · DOI · Europe PMC
  • 2026-09开放获取
    High Resolution 3-T Magnetic Resonance Tractography Cannot Localise Conduction Block in Multifocal Motor Neuropathy With Conduction Block
    Journal of the peripheral nervous system : JPNS · DOI · Europe PMC
  • 2026-09开放获取
    Unexpected Conduction Blocks in <i>SORD</i>-Related Distal Motor Neuropathy: A Case Report
    Clinical case reports · DOI · Europe PMC
  • 2026-09
    Spectrum of Hereditary Neuropathies in Adult Patients From Serbia
    Journal of the peripheral nervous system : JPNS · DOI · Europe PMC
  • 2026-08荟萃分析系统综述开放获取
    Effects of therapeutic plasma exchange (TPE) on efficacy and adverse events in patients with autoimmune neurological diseases: a systematic review and meta-analysis based on real-world studies and single-arm clinical trials
    Frontiers in neurology · DOI · Europe PMC
  • 2026-08综述开放获取
    Magnetic resonance imaging applications to amyotrophic lateral sclerosis beyond the central nervous system
    Frontiers in neurology · DOI · Europe PMC
  • 2026-08综述开放获取
    A cardiovascular-motor axis framework for perfusion-mediated motor impairment
    International journal of cardiology. Heart & vasculature · DOI · Europe PMC
  • 2026-08
    Hyaluronidase-Facilitated Subcutaneous Immunoglobulin 10% as Maintenance Therapy for Japanese Patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy and Multifocal Motor Neuropathy: A Phase 3, Open-label Clinical Trial
    Internal medicine (Tokyo, Japan) · DOI · Europe PMC
  • 2026-08综述开放获取
    Diffusion-Weighted Imaging in the Musculoskeletal System: Evolving Role in Modern Imaging Practice
    Diagnostics (Basel, Switzerland) · DOI · Europe PMC
  • 2026-08病例报告开放获取
    An Unusual Iatrogenic Complication of Botulinum Toxin: Forearm Flexor Weakness Following a Nonstandard Intradermal Sensitivity Test
    Aesthetic surgery journal. Open forum · DOI · Europe PMC
  • 2026-08综述开放获取
    The Role of Neurofilaments in Diagnosis and Monitoring of Amyotrophic Lateral Sclerosis
    Journal of clinical medicine · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Ganglioside Antibodies as Formidable Neurologic Mimickers: A Case Report of Dual Anti-GQ1b and Asialo-GM1 Antibody Syndrome
    Cureus · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Bilateral Parsonage-Turner Syndrome in a Young Adult Man Due to Parvovirus B19 Infection
    Cureus · DOI · Europe PMC
  • 2026-08开放获取
    Neuralgic amyotrophy - Beyond the plexus: A case of surgical treatment of bilateral distal peripheral nerve involvement in neuralgic amyotrophy/Parsonage-Turner Syndrome
    Brain & spine · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Cladribine treatment in chronic inflammatory demyelinating polyradiculoneuropathy and multifocal motor neuropathy: two case reports
    Frontiers in immunology · DOI · Europe PMC
  • 2026-08综述开放获取
    MyD88-Family Adaptors: Compartmentalised Signalling and Non-Immune Functions
    Immunology · DOI · Europe PMC
  • 2026-08开放获取
    Refining Electrophysiological Criteria in CIDP and MMN: Toward a Unified Definition of Motor Conduction Block and Proximal Temporal Dispersion
    European journal of neurology · DOI · Europe PMC

境外已获批用于本病的药物 1L2

欧盟 0 项、美国 1 项。同一药物在两地各批一次的,会分别列出。

「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径,各有各的条件与费用。这一节能确定地告诉你的只有一件事:这个病在世界范围内已经有获得批准的药物,它叫什么名字。拿这个名字去问主治医生,是下一步最省力的做法。

药名一律保留英文原文,不作翻译——中国的药品通用名与英文名的音译经常不一致,译错会让人去找一个不存在的药。

  • Gammagard Liquid美国2012-06-22
    immune globulin infusion (human)
    官方记录
尚未获批的在研药物(2 项)

这些药已被欧盟或美国的监管机构认定为罕见病用药(英文 orphan drug designation,中文行业里通称「孤儿药资格」——「孤儿」说的是这类药市场太小、没有厂商愿意认领,不是在说病人)。但这只是一种监管身份:意味着监管机构给予研发上的激励,不代表这个药已被证明有效,也不代表将来一定能上市,绝大多数最终不会成药。列在这里是为了看清有哪些方向正在被尝试。

  • empasiprubart欧盟2025-06-20
    Treatment of multifocal motor neuropathy
    官方记录
  • empasiprubart美国2024-06-05
    treatment of multifocal motor neuropathy
    官方记录

数据来自欧洲药品管理局(EMA)的药品与罕见病用药资格公开导出表,以及美国 FDA 的罕见病用药资格数据库(Orphan Drug Product Designation Database)。两边口径不同:欧盟一侧取的是当前状态仍为「已授权」的药品;美国一侧记录的是「曾获批准」这一事实,FDA 的公开表不追踪药物此后是否退市(例如 Relyvrio 于 2024 年撤市,表中仍记为已获批)。请以官方记录页为准。

在中国开展的临床试验 3L2

按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实,并与主治医生商量。

当前没有检索到登记为可入组的试验。

其他状态的试验(3 项)
  • 进行中·不再招募NCT05988073
    A Prospective Longitudinal Study in Adults With Multifocal Motor Neuropathy
    观察性 · 2023/11/29argenx
    中国研究中心 3 个:Changsha、Nanchang、Shanghai
  • 进行中·不再招募NCT06537999
    A Clinical Study to Evaluate DNTH103 in Adults With Multifocal Motor Neuropathy
    II 期 · 干预性 · 2024/09/17Dianthus Therapeutics
    中国研究中心 3 个:Beijing、Shanghai
  • 进行中·不再招募NCT06742190
    A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy
    III 期 · 干预性 · 2024/12/18argenx
    中国研究中心 1 个:Shanghai

中国境外的在招试验 2L2

这些试验在中国没有研究中心,通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处:看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生,或据此进一步查该药是否已在境外获批、是否有拓展性用药(expanded access)通道。

Georgia1澳大利亚1美国1

共 2 项。

  • 招募中NCT07610564
    A Study to Assess the Effect of Empasiprubart on Nerve Morphology by Ultrasound in Adults With Multifocal Motor Neuropathy
    I 期 · 干预性 · 2026/07/27argenx
    Georgia
  • 招募中NCT01793168
    Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
    观察性 · 2010/07Sanford Health
    澳大利亚、美国

外部标识与链接

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本页数据来源

  • 疾病定义、同义词、基因、表型、流行病学:Orphanet(CC BY 4.0)
  • 中文病名:Orphanet 中文包,冻结于 2020-06-01
  • 表型中文标签:HPO 简体中文翻译(CHPO 上游成果)