RARE DISEASE
Lambert-Eaton myasthenic syndrome
Lambert-Eaton myasthenic syndrome
Lambert-Eaton myasthenic syndrome
Drug discovery
5
drugs
With orphan designations
Overview
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder targeting presynaptic voltage-gated calcium channels at the neuromuscular junction, impairing acetylcholine release and causing proximal muscle weakness, hyporeflexia, and autonomic dysfunction [1][6][7]. Approximately 50-60% of cases are paraneoplastic, typically associated with small-cell lung cancer (SCLC) [1][6]. Diagnosis involves antibody testing, electromyography, and cancer screening [7][15].
Burden
Health-related quality of life (HRQoL): Significantly lower than in myasthenia gravis and the general population, with impaired physical function (SF-36 score 34.2 vs. 78.6 in genP) [5][9][13].
Diagnostic delays: 58% initially misdiagnosed, often as myasthenia gravis or depression [10][12].
Disability: 25% require wheelchair use; prolonged disease duration in AI-LEMS (median 7.5 years) [10][16].
Therapies
First-line: 3,4-diaminopyridine (amifampridine) to enhance neuromuscular transmission [3][15].
Immunotherapy: IVIG, plasma exchange, or immunosuppressants (e.g., prednisone, azathioprine) for refractory cases [8][14].
Paraneoplastic: Tumor-directed therapy (e.g., chemotherapy for SCLC) improves LEMS outcomes [1][19].
Categories: rare neurological diseases
Research Papers
332 drug discovery papers about Lambert-Eaton myasthenic syndrome, with 1 first-in-class and 7 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
332 drug discovery papers about Lambert-Eaton myasthenic syndrome, with 1 first-in-class and 7 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
small molecules
2026-08-17 | Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS.
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular junction disorder characterized by muscle weakness and autonomic dysfunction. While amifampridine is the established first-line symptomatic treatment, longitudinal data regarding the evolution of therapeutic regimens over time and the use of immunosuppressive therapies remain limited. This retrospective study analyzed 70 LEMS patients treated at the Leiden University Medical Center (LUMC) between 1992 and 2023. Treatment was evaluated and a four-point "leg score" was developed and used to retrospectively quantify functional impairment and response to amifampridine. Sixty-six percent of the cohort was female, the median age of diagnosis was 54 years and non-tumor LEMS was more prevalent (77%). All patients used amifampridine at some point, and 91% initiated it within six months of diagnosis. Amifampridine improved leg scores in 57% of patients. Although 58% of patients reported side effects (mostly paresthesia), discontinuation was rare (7%). Pyridostigmine was used in 81% of patients. Immunosuppressive drug use-predominantly prednisolone and azathioprine -increased over time, reaching 44% after five years. Conversely, azathioprine, prednisolone and pyridostigmine were discontinued by 50%, 38% and 32% of patients, respectively, often due to adverse effects. In conclusion, amifampridine provides significant clinical benefits and remains the cornerstone of LEMS therapy. Immunomodulatory therapies were introduced gradually and their use increased over time. Compared with autoimmune myasthenia gravis, immunomodulatory therapy was used less frequently during the early disease course of LEMS.
2026-07-16 | Clinical approach to neurological paraneoplastic syndromes.
Neurological paraneoplastic syndromes are immune-mediated disorders. The diagnosis of paraneoplastic syndromes is often difficult because they are rare and the onset of symptoms usually precedes detection of the underlying malignancy. Accurate diagnosis depends on recognition of the clinical phenotypes likely to be associated with a paraneoplastic aetiology and detection of an anti-neuronal antibody. Clinical phenotypes associated with a high risk of a paraneoplastic cause are limbic encephalitis, encephalomyelitis, rapidly progressive cerebellar syndrome, opsoclonus-myoclonus syndrome, subacute sensory neuronopathy, enteric neuropathy and Lambert Eaton myasthenic syndrome. Clinical phenotypes with an intermediate risk of a paraneoplastic aetiology are brainstem encephalitis, myelopathy, stiff person syndrome, Morvan syndrome and retinopathy. Anti-neuronal antibodies are extremely useful in the diagnosis of paraneoplastic neurological syndromes, but false-negative results can occur when only serum or cerebrospinal fluid (CSF) is tested. Commercial antibody panels do not include all antibodies associated with paraneoplastic syndromes, and if this diagnosis is strongly suspected, serum and CSF should be sent to a research laboratory. The risk of a false-positive result is increased if antibody tests are requested when there is a low pre-test probability of a paraneoplastic syndrome or if an antibody is detected in low titre.
2026-07-01 | Physiotherapy Management of an Older Adult with LambertEaton Myasthenic Syndrome Presenting with Proximal Limb Weakness and Autonomic Dysfunction: A Case Report
Introduction: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune presynaptic disorder of the neuromuscular junction, with an estimated prevalence of approximately 2.6 to 10 cases per million population.1,2 It is frequently under-recognized in physiotherapy settings because its insidious, fatigable proximal weakness can mimic deconditioning, lumbar radiculopathy, or age-related sarcopenia in older adults. This report describes the physiotherapy assessment and rehabilitation of a 60-year-old retired male with autoimmune LEMS, with particular attention to the use of low-intensity, fatigue-monitored exercise progression — an approach with limited prior documentation in the physiotherapy literature, as no controlled trials of physiotherapy specifically in LEMS currently exist. Case presentation: A 60-year-old retired male presented with a six-month history of progressive bilateral proximal lower limb weakness, difficulty rising from a chair and climbing stairs, dry mouth, and mild ptosis that worsened through the day and partially recovered with brief exertion. Tendon reflexes were diminished but augmented following sustained contraction. Management and outcomes: Diagnosis was confirmed by repetitive nerve stimulation showing post-tetanic facilitation and positive P/Q-type voltage-gated calcium channel (VGCC) antibodies, with no malignancy detected on staging investigations. Medical management with pyridostigmine and graded immunotherapy was combined with a physiotherapy programme emphasising submaximal aerobic and resistance exercise, energy conservation education, balance retraining, and fatigue self-monitoring. Over 12 weeks, the patient demonstrated improved manual muscle testing grades, a clinically meaningful reduction in Timed Up and Go time, and improved scores on the Myasthenia Gravis Quality of Life scale, without provoking overwork weakness. Conclusion: Individually tailored, fatigue-monitored physiotherapy can be safely incorporated alongside immunomodulatory and symptomatic pharmacological treatment in LEMS, supporting functional independence in an older adult. Close collaboration between neurology and physiotherapy, with vigilance for overwork weakness and underlying malignancy, is essential.
2026-05-20 | Nanotechnology-driven therapeutic strategies for lambert–eaton myasthenic syndrome: Emerging drug delivery and immunomodulatory approaches
Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular disorder characterized by impaired acetylcholine release due to autoantibody-mediated targeting of presynaptic P/Q-type voltage-gated calcium channels. Although symptomatic therapies such as amifampridine and immunomodulatory agents provide clinical benefit, their efficacy is limited by short half-life, fluctuating plasma concentrations, systemic toxicity, and dose-dependent adverse effects, including seizures. These limitations highlight the need for advanced drug delivery systems. Recent advances in nanotechnology offer promising strategies to enhance therapeutic precision, bioavailability, and safety in LEMS management. Nanoparticle-based platforms including polymeric nanoparticles and lipid-based nanocarriers enable controlled drug release and improved pharmacokinetic stability, which may help maintain sustained plasma concentrations and thereby enhance drug availability at the neuromuscular junction. In addition, nanocarriers may facilitate improved transport across physiological barriers such as the Blood Nerve Barrier, potentially increasing drug accumulation at presynaptic terminals. Intranasal nanoformulations are explored as a non-invasive systemic delivery strategy, leveraging the highly vascularized nasal mucosa to enhance drug absorption and bypass hepatic first-pass metabolism, thereby improving systemic exposure rather than enabling direct central nervous system targeting. Furthermore, nano-enabled immunomodulation may allow selective suppression of pathogenic autoantibodies while minimizing systemic immunosuppression. This review provides a comprehensive overview of LEMS pathophysiology and current therapies, with a focused discussion on nanotechnology-driven approaches that may overcome existing pharmacological limitations. Future perspectives in targeted delivery, nano-immunotherapy, and biomarker-guided treatment strategies are also highlighted.
2026-04-29 | Lambert-Eaton Myasthenic Syndrome During Immunotherapy in Extensive-Stage Small-Cell Lung Cancer: A Case Report.
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder of the neuromuscular junction that is frequently associated with small-cell lung cancer (SCLC). It is characterized by proximal muscle weakness, reduced deep tendon reflexes, and autonomic symptoms, all of which can significantly impair quality of life and complicate oncologic management. Early recognition and timely intervention are crucial to improving clinical outcomes. We report a case of a 76-year-old man with extensive-stage SCLC and chronic renal insufficiency whose LEMS worsened during durvalumab immunotherapy. Despite continued chemotherapy and immunotherapy, his symptoms progressively worsened. Chemotherapy failed to improve his neurological condition, with subsequent recurrent episodes of muscle weakness. Subsequent administration of intravenous immunoglobulin (IVIG) combined with pyridostigmine led to marked neurological improvement and restoration of muscle strength to grade 5, enabling continuation of antitumor therapy. The patient subsequently received three cycles of second-line lurbinectedin chemotherapy, during which inflammatory responses, anemia, and venous thrombosis developed but were effectively managed with supportive treatment. Following chemotherapy and immunotherapy, the patient's limb muscle strength recovered to grade 5, followed by recurrent muscle weakness symptoms. Hematologic and biochemical parameters gradually normalized, and inflammatory markers returned to baseline. At discharge, his clinical condition was stable. This case highlights the importance of early recognition and standardized management of paraneoplastic LEMS in enhancing functional recovery and enabling the safe continuation of antitumor therapy.
cell therapies
2026-04-24 | Cellular therapies in autoimmune neuromuscular disorders: the new frontier.
Antibody-mediated humoral immunity plays a vital role in a number of autoimmune neuromuscular disorders (AINMD), such as myasthenia gravis, Lambert-Eaton myasthenic syndrome (LEMS), stiff-person syndrome (SPS), and autoimmune myositides. Although biologics based B cell and plasma cell depletion (BCDT) therapy effectively treats some AINMDs, its variable success highlights the limitations of this therapeutic modality. B cell directed cellular therapy (BCDcT) targeting a wide array of B cell lineages, including short-lived plasma cells, has emerged as an effective treatment modality in hematological malignancies, resulting in impressive reductions in tumor burden, relapse rates, and mortality. Despite the impressive success of BCDcT, especially in hematological malignancies, its initial use was limited to last-resort cases due to the high morbidity associated with cytokine release syndrome (CRS) and immune cell-associated neurological toxicity (ICANS). Recent improvements in the safety profile of BCDcTs and better clinical management have lowered the incidences of CRS and ICANS, leading to its earlier use in some cancers. The success of BCDcT in B cell malignancies has promoted strong interest in applying this therapeutic approach to autoimmune disorders, particularly AINMD. This review will cover cell therapy fundamentals, limitations of current BCDTs, experiences with cell therapy in AINMD, and both ongoing and planned trials in this field.
2026-03-13 | CAR T cell therapy in autoantibody-mediated neurological disorders: a promising strategy.
Chimeric antigen receptor (CAR) T cell therapy is emerging as a promising approach for B cell-driven neurological autoimmune disorders, particularly those characterized by pathogenic autoantibodies that target key neural structures. These conditions, including neuromyelitis optica spectrum disorder, myasthenia gravis, Lambert-Eaton myasthenic syndrome, MOG antibody-associated disease, anti-NMDA receptor encephalitis, Diacylglycerol lipase alpha antibody associated encephalitis, stiff person syndrome, and multiple sclerosis, can be categorized based on their primary autoantigens into (1) extracellular antigen-associated (e.g., AQP4, AChR, NMDAR, MOG, VGCC), (2) intracellular antigen-associated (e.g., GAD65, DAGLA), or (3) unidentified antigenic origin (as seen in multiple sclerosis). This distinction is essential for guiding therapeutic strategies and exploring novel principles represented in distinct treatment approaches and their corresponding therapeutic outcomes. In this review, we propose a classification of CAR T cell therapies designed for different target antigens, including: CD19/20/BCMA-directed CAR T cells targeting general B cell-mediated pathogenesis, regulatory T cells modified with CARs, and the design of chimeric autoantibody receptors (CAARs) to selectively deplete pathogenic B cells directly associated with disease progression while preserving immune tolerance. We further discuss preclinical and clinical advancements, key challenges such as safety concerns and neurotoxicity, and the future landscape of CAR T applications in neuromyelitis optica spectrum disorder, myasthenia gravis, Lambert-Eaton myasthenic syndrome, MOG antibody-associated disease, anti-NMDA receptor encephalitis, Diacylglycerol lipase alpha antibody associated encephalitis, stiff person syndrome, and multiple sclerosis according to the latest research, case and trial data. CAR T cell therapy potentially offers a highly specific and effective method with thorough elimination of autoreactive B cells, representing a rapidly evolving field with the potential to transform the treatment of autoimmune neurological disorders. As CAR T technology advances, it holds the potential to become a groundbreaking immunoablative strategy with so-far disclosed controllable side effects; however, further long-term follow-up data are still needed to validate its application in autoimmune disorders.
2025-06-27 | A Rare Case of Lambert-Eaton Myasthenia Syndrome With Dysphasia and Dysarthria.
Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder resulting in muscle weakness. LEMS typically presents with progressive proximal muscle weakness, especially in the lower extremities, areflexia, and autonomic dysfunction. Bulbar involvement and respiratory compromise tend to occur later in the disease course and usually in severe, advanced cases. We report a unique case of a 50-year-old male with a known history of LEMS, autoimmune retinopathy, erectile dysfunction, and prediabetes who presented with progressive bulbar symptoms - slurred speech and difficulty swallowing - without accompanying weakness in the upper or lower extremities. Despite receiving four doses of intravenous immunoglobulin (IVIG) prior to admission with no improvement, he showed a significant clinical response to six sessions of plasmapheresis. Notably, the absence of limb weakness and preserved motor strength throughout his hospital stay raised diagnostic uncertainty, prompting evaluation for myasthenia gravis, which was ruled out through negative acetylcholine receptor antibody testing and reaffirmed voltage-gated calcium channel (VGCC) antibody positivity. Chest imaging showed no underlying malignancy. This case illustrates an atypical LEMS presentation dominated by severe bulbar dysfunction in the absence of limb involvement or respiratory failure, which is uncommon and may mimic other neuromuscular junction disorders such as myasthenia gravis. The favorable response to plasmapheresis emphasizes the importance of early recognition and aggressive immunomodulatory treatment in preventing clinical deterioration. Clinicians should maintain a high index of suspicion for LEMS even in unusual presentations to ensure timely and appropriate management.
2024-10-25 | Anti-CD19 CAR-T cells are effective in severe idiopathic Lambert-Eaton myasthenic syndrome.
Lambert-Eaton myasthenic syndrome (LEMS) is an autoantibody-mediated disease of the neuromuscular junction characterized by muscular weakness. Autoantibodies to presynaptic P/Q-type voltage-gated calcium channels (VGCCs) induce defective neuromuscular function. In severe cases, current immunosuppressive and immunomodulatory treatment strategies are often insufficient. First reports show beneficial effects of anti-CD19 chimeric antigen receptor (CAR)-T cell therapy in patients with autoantibody-mediated myasthenia gravis. We report a patient with isolated idiopathic LEMS treated with autologous anti-CD19-CAR-T cells. In this patient, CAR-T infusion leads to expansion of predominantly CD4+ CAR-T cells with a terminally differentiated effector memory cells re-expressing CD45RA (TEMRA)-like phenotype indicating cytotoxic capabilities and subsequent B cell depletion. VGCC antibody titers decrease, resulting in a clinical improvement of LEMS symptoms, e.g., 8-fold increase in walking distance. The patient does not show relevant side effects except for cytokine release syndrome grade 2 and intermittent neutropenia suggesting that anti-CD19 CAR-T cell therapy may be a treatment option in patients with LEMS.
2024-06-06 | Treatment of concomitant myasthenia gravis and Lambert-Eaton myasthenic syndrome with autologous CD19-targeted CAR T cells.
Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR T cells for treating concomitant MG/LEMS. After CAR T cell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage-gated calcium channel N-type autoantibody levels paralleled major neurological responses. Within 2 months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4 months post-CAR T cell infusion, respectively. This report highlights the potential for anti-CD19 CAR T cells to achieve profound clinical effects in the treatment of neuroimmunological diseases.
antibodies
2026-01-24 | Clinical Spectrum and Outcomes of SOX1 Antibody-Associated Paraneoplastic Neurological Syndromes: A Chinese Cohort Study.
SOX1 antibody-positive paraneoplastic neurological syndromes (PNS) exhibit significant population-specific clinical heterogeneity. While Western cohorts predominantly manifest Lambert-Eaton myasthenic syndrome (65%-80%), comprehensive clinical characterization and treatment response data in Asian populations remain critically limited. We conducted a single-center retrospective case series analyzing 13 consecutive patients with SOX1 antibody-positive PNS treated at Guangdong Sanjiu Brain Hospital from January 2019 to December 2024. SOX1 antibodies were confirmed using commercial immunoblot assay. Primary endpoints included treatment response (≥ 1-point improvement on modified Rankin Scale [mRS]) and functional recovery (mRS ≤ 2). Statistical analyses employed Fisher's exact tests and Mann-Whitney U tests. Among 13 patients (median age 61 years [IQR 56-67], 53.8% female), neuropsychiatric presentations predominated, including seizures (46.2%) and psychiatric symptoms (30.8%), with combined neuropsychiatric manifestations occurring in 53.8% of patients. Co-existing neuronal antibodies were identified in 15.4% of cases (GABAB receptor, LGI1). Malignancy was confirmed in 30.8% of patients. Immunotherapy recipients (n = 7) demonstrated significantly superior functional outcomes compared to non-treated patients: median 3-month mRS 0 (IQR 0-0) versus 3 (IQR 3-3), p = 0.03. Treatment response rates were 85.7% versus 33.3% (p = 0.103). Chinese patients with SOX1 antibody-positive PNS demonstrate a neuropsychiatric-predominant phenotype (53.8%), contrasting markedly with Western cohorts. Early immunotherapy administration was associated with superior functional outcomes (median 3-month mRS: 0 vs. 3, p = 0.03). These findings support comprehensive neuronal antibody profiling and early immunotherapy consideration in patients presenting with neuropsychiatric manifestations.
2026-01-08 | Coexisting cerebellar ataxia and Lambert-Eaton myasthenic syndrome without malignancy: insights from a case and systematic literature review.
BACKGROUND: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder often associated with small-cell lung carcinoma (SCLC). Cerebellar degeneration can coexist with LEMS, although their simultaneous presentation in the non-neoplastic setting is uncommon. METHODS: We describe a 75-year-old man with progressive cerebellar ataxia followed by bilateral ptosis, ophthalmoparesis, and proximal weakness. LEMS was confirmed by positive P/Q-type VGCC antibodies and electrophysiology. Extensive malignancy screening, including PET-CT, was negative, and immunotherapy produced marked neuromuscular and partial cerebellar improvement. To contextualize this case, we systematically reviewed the published literature (January 1991- August 2025) for this association, including cohorts, case reports and series providing individual patient-level details. RESULTS: We identified 37 studies reporting 67 patients with coexistent LEMS and cerebellar ataxia. Malignancy was present in 71.6% (48/67), predominantly SCLC (35/67, 52.2%). The median interval from neurological symptom onset to cancer diagnosis was 3 months (IQR 0.5–6.0; range − 60 to 39 months). Anti-VGCC antibodies were detected in 83.6% (56/67), anti-SOX1 in 7.5% (5/67), and anti-Hu in 3.0% (2/67), while 6.0% (4/67) were seronegative. Notably, 28.4% (19/67) had no malignancy during follow-up, suggesting a non-paraneoplastic autoimmune subset. Neurological improvement was comparable between paraneoplastic and non-paraneoplastic cases, but mortality was significantly higher in malignancy-associated cases (27.1% vs. 5.3%, p = 0.04). CONCLUSION: Coexistent LEMS and cerebellar ataxia is not invariably paraneoplastic. Early immunotherapy may improve neurological outcomes even in cancer-negative cases, but long-term oncologic surveillance remains essential.
2025-12-03 | Case Report: A Patient with Lambert-Eaton Myasthenic Syndrome Successfully Treated with Efgartigimod.
Lambert-Eaton myasthenic syndrome (LEMS) is a neuromuscular junction disorder which is frequently associated with tumors. Those patients generate pathogenic antibodies such as voltage-gated calcium channel (VGCC) antibodies. Existing studies have established the efficacy of efgartigimod in MG. This article reports the effect of efgartigimod following its administration to a patient with LEMS. A 73-year-old male was diagnosed with small cell lung cancer two years ago. Gradually he developed difficulties in speaking, eating, and lifting his head, along with limb weakness. The neuroelectrophysiological examination findings were consistent with LEMS. Laboratory tests detected VGCC antibodies and SOX-1 antibodies in the serum. The patient's symptoms continued to worsen after one week of corticosteroids use. Consequently, efgartigimod at a dose of 10 mg/kg was added once a week. After three weeks of treatment, the patient's clinical manifestations had improved significantly. Laboratory tests revealed a decrease in serum VGCC antibody and IgG levels. This is the first report on the use of efgartigimod in LEMS. The patient demonstrated substantial clinical improvement following treatment. Nevertheless, more clinical studies are required to verify whether efgartigimod offers a novel treatment approach for patients with LEMS.
2025-11-26 | Lambert-Eaton myasthenic syndrome presenting with occult mediastinal small cell carcinoma and positivity for anti-CV2/CRMP5 and anti-SOX1 antibodies: a case report.
BACKGROUND: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular disorder characterized by proximal muscle weakness, autonomic dysfunction and hyporeflexia. Numerous studies have reported the association of LEMS with anti-SOX1 antibodies and small cell carcinoma, but LEMS with double antibody positivity and cerebellar ataxia accompanied by small cell carcinoma is relatively rare. CASE PRESENTATION: A 56-year-old female patient developed progressive limb weakness in November 2023, with symptoms gradually worsening over six months to include gait instability, impaired grip, chest tightness, dyspnea, dysphagia, and aspiration. Electrodiagnostic testing confirmed Lambert-Eaton myasthenic syndrome (LEMS) with positive VGCC antibodies. Paraneoplastic antibody testing showed positive anti-CV2/CRMP5 antibodies (24 AU) and borderline anti-SOX1 antibodies (9 AU), while other antibodies were negative. Initial imaging (contrast-enhanced chest CT and PET-CT) revealed no abnormalities, but follow-up one year later detected mediastinal small cell lung cancer. The patient underwent three plasma exchange sessions with corticosteroid therapy with limited response, then showed significant improvement after switching to subcutaneous Telitacicept (80 mg) combined with Fampyra Comprimidos and Mycophenolate Mofetil capsules, achieving independent ambulation and normal grip strength with only mild residual limb weakness. The patient has since been transferred to a local hospital for chemotherapy. CONCLUSION: We find that LEMS with concurrent anti-SOX1 and anti-CV2/CRMP5 positivity manifests as rapid progression, prominent proximal weakness, and potential cerebellar and autonomic involvement. Early and sustained oncological surveillance—particularly of mediastinal lymph nodes—and combined immunotherapeutic strategies can significantly improve neurological outcomes.
2025-01-22 | Association of Lambert-Eaton Myasthenic Syndrome and First Episode Psychosis: A Case Report.
Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune neuromuscular junction disorder characterized by proximal weakness, autonomic dysfunction, and areflexia associated with antibodies against voltage-gated calcium channels (VGCCs). Psychotic symptoms can occur in many autoimmune neurological disorders but they have rarely been observed in myasthenic syndromes. We report the case of a 21-year-old woman with primary autoimmune LEMS due to anti-VGCC antibodies subtype P/Q, who developed psychotic symptoms 3 years after the onset of motor symptoms. These symptoms decreased after every cycle of monthly intravenous immunoglobulin therapy. Different causes of reversible psychosis were excluded, such as autoimmune encephalitis. Due to a worsening of the patient's muscle strength, and the psychotic episodes, the patient received several treatments including an admission to a neurology unit. To our knowledge, this is the first described case of psychotic symptoms associated with LEMS. We speculate that VGCC antibodies could have a role in the development of mental symptoms, although additional hypotheses are also discussed.
small molecules
2026-08-17 | Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS.
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular junction disorder characterized by muscle weakness and autonomic dysfunction. While amifampridine is the established first-line symptomatic treatment, longitudinal data regarding the evolution of therapeutic regimens over time and the use of immunosuppressive therapies remain limited. This retrospective study analyzed 70 LEMS patients treated at the Leiden University Medical Center (LUMC) between 1992 and 2023. Treatment was evaluated and a four-point "leg score" was developed and used to retrospectively quantify functional impairment and response to amifampridine. Sixty-six percent of the cohort was female, the median age of diagnosis was 54 years and non-tumor LEMS was more prevalent (77%). All patients used amifampridine at some point, and 91% initiated it within six months of diagnosis. Amifampridine improved leg scores in 57% of patients. Although 58% of patients reported side effects (mostly paresthesia), discontinuation was rare (7%). Pyridostigmine was used in 81% of patients. Immunosuppressive drug use-predominantly prednisolone and azathioprine -increased over time, reaching 44% after five years. Conversely, azathioprine, prednisolone and pyridostigmine were discontinued by 50%, 38% and 32% of patients, respectively, often due to adverse effects. In conclusion, amifampridine provides significant clinical benefits and remains the cornerstone of LEMS therapy. Immunomodulatory therapies were introduced gradually and their use increased over time. Compared with autoimmune myasthenia gravis, immunomodulatory therapy was used less frequently during the early disease course of LEMS.
2026-07-16 | Clinical approach to neurological paraneoplastic syndromes.
Neurological paraneoplastic syndromes are immune-mediated disorders. The diagnosis of paraneoplastic syndromes is often difficult because they are rare and the onset of symptoms usually precedes detection of the underlying malignancy. Accurate diagnosis depends on recognition of the clinical phenotypes likely to be associated with a paraneoplastic aetiology and detection of an anti-neuronal antibody. Clinical phenotypes associated with a high risk of a paraneoplastic cause are limbic encephalitis, encephalomyelitis, rapidly progressive cerebellar syndrome, opsoclonus-myoclonus syndrome, subacute sensory neuronopathy, enteric neuropathy and Lambert Eaton myasthenic syndrome. Clinical phenotypes with an intermediate risk of a paraneoplastic aetiology are brainstem encephalitis, myelopathy, stiff person syndrome, Morvan syndrome and retinopathy. Anti-neuronal antibodies are extremely useful in the diagnosis of paraneoplastic neurological syndromes, but false-negative results can occur when only serum or cerebrospinal fluid (CSF) is tested. Commercial antibody panels do not include all antibodies associated with paraneoplastic syndromes, and if this diagnosis is strongly suspected, serum and CSF should be sent to a research laboratory. The risk of a false-positive result is increased if antibody tests are requested when there is a low pre-test probability of a paraneoplastic syndrome or if an antibody is detected in low titre.
2026-07-01 | Physiotherapy Management of an Older Adult with LambertEaton Myasthenic Syndrome Presenting with Proximal Limb Weakness and Autonomic Dysfunction: A Case Report
Introduction: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune presynaptic disorder of the neuromuscular junction, with an estimated prevalence of approximately 2.6 to 10 cases per million population.1,2 It is frequently under-recognized in physiotherapy settings because its insidious, fatigable proximal weakness can mimic deconditioning, lumbar radiculopathy, or age-related sarcopenia in older adults. This report describes the physiotherapy assessment and rehabilitation of a 60-year-old retired male with autoimmune LEMS, with particular attention to the use of low-intensity, fatigue-monitored exercise progression — an approach with limited prior documentation in the physiotherapy literature, as no controlled trials of physiotherapy specifically in LEMS currently exist. Case presentation: A 60-year-old retired male presented with a six-month history of progressive bilateral proximal lower limb weakness, difficulty rising from a chair and climbing stairs, dry mouth, and mild ptosis that worsened through the day and partially recovered with brief exertion. Tendon reflexes were diminished but augmented following sustained contraction. Management and outcomes: Diagnosis was confirmed by repetitive nerve stimulation showing post-tetanic facilitation and positive P/Q-type voltage-gated calcium channel (VGCC) antibodies, with no malignancy detected on staging investigations. Medical management with pyridostigmine and graded immunotherapy was combined with a physiotherapy programme emphasising submaximal aerobic and resistance exercise, energy conservation education, balance retraining, and fatigue self-monitoring. Over 12 weeks, the patient demonstrated improved manual muscle testing grades, a clinically meaningful reduction in Timed Up and Go time, and improved scores on the Myasthenia Gravis Quality of Life scale, without provoking overwork weakness. Conclusion: Individually tailored, fatigue-monitored physiotherapy can be safely incorporated alongside immunomodulatory and symptomatic pharmacological treatment in LEMS, supporting functional independence in an older adult. Close collaboration between neurology and physiotherapy, with vigilance for overwork weakness and underlying malignancy, is essential.
2026-05-20 | Nanotechnology-driven therapeutic strategies for lambert–eaton myasthenic syndrome: Emerging drug delivery and immunomodulatory approaches
Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular disorder characterized by impaired acetylcholine release due to autoantibody-mediated targeting of presynaptic P/Q-type voltage-gated calcium channels. Although symptomatic therapies such as amifampridine and immunomodulatory agents provide clinical benefit, their efficacy is limited by short half-life, fluctuating plasma concentrations, systemic toxicity, and dose-dependent adverse effects, including seizures. These limitations highlight the need for advanced drug delivery systems. Recent advances in nanotechnology offer promising strategies to enhance therapeutic precision, bioavailability, and safety in LEMS management. Nanoparticle-based platforms including polymeric nanoparticles and lipid-based nanocarriers enable controlled drug release and improved pharmacokinetic stability, which may help maintain sustained plasma concentrations and thereby enhance drug availability at the neuromuscular junction. In addition, nanocarriers may facilitate improved transport across physiological barriers such as the Blood Nerve Barrier, potentially increasing drug accumulation at presynaptic terminals. Intranasal nanoformulations are explored as a non-invasive systemic delivery strategy, leveraging the highly vascularized nasal mucosa to enhance drug absorption and bypass hepatic first-pass metabolism, thereby improving systemic exposure rather than enabling direct central nervous system targeting. Furthermore, nano-enabled immunomodulation may allow selective suppression of pathogenic autoantibodies while minimizing systemic immunosuppression. This review provides a comprehensive overview of LEMS pathophysiology and current therapies, with a focused discussion on nanotechnology-driven approaches that may overcome existing pharmacological limitations. Future perspectives in targeted delivery, nano-immunotherapy, and biomarker-guided treatment strategies are also highlighted.
2026-04-29 | Lambert-Eaton Myasthenic Syndrome During Immunotherapy in Extensive-Stage Small-Cell Lung Cancer: A Case Report.
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder of the neuromuscular junction that is frequently associated with small-cell lung cancer (SCLC). It is characterized by proximal muscle weakness, reduced deep tendon reflexes, and autonomic symptoms, all of which can significantly impair quality of life and complicate oncologic management. Early recognition and timely intervention are crucial to improving clinical outcomes. We report a case of a 76-year-old man with extensive-stage SCLC and chronic renal insufficiency whose LEMS worsened during durvalumab immunotherapy. Despite continued chemotherapy and immunotherapy, his symptoms progressively worsened. Chemotherapy failed to improve his neurological condition, with subsequent recurrent episodes of muscle weakness. Subsequent administration of intravenous immunoglobulin (IVIG) combined with pyridostigmine led to marked neurological improvement and restoration of muscle strength to grade 5, enabling continuation of antitumor therapy. The patient subsequently received three cycles of second-line lurbinectedin chemotherapy, during which inflammatory responses, anemia, and venous thrombosis developed but were effectively managed with supportive treatment. Following chemotherapy and immunotherapy, the patient's limb muscle strength recovered to grade 5, followed by recurrent muscle weakness symptoms. Hematologic and biochemical parameters gradually normalized, and inflammatory markers returned to baseline. At discharge, his clinical condition was stable. This case highlights the importance of early recognition and standardized management of paraneoplastic LEMS in enhancing functional recovery and enabling the safe continuation of antitumor therapy.
cell therapies
2026-04-24 | Cellular therapies in autoimmune neuromuscular disorders: the new frontier.
Antibody-mediated humoral immunity plays a vital role in a number of autoimmune neuromuscular disorders (AINMD), such as myasthenia gravis, Lambert-Eaton myasthenic syndrome (LEMS), stiff-person syndrome (SPS), and autoimmune myositides. Although biologics based B cell and plasma cell depletion (BCDT) therapy effectively treats some AINMDs, its variable success highlights the limitations of this therapeutic modality. B cell directed cellular therapy (BCDcT) targeting a wide array of B cell lineages, including short-lived plasma cells, has emerged as an effective treatment modality in hematological malignancies, resulting in impressive reductions in tumor burden, relapse rates, and mortality. Despite the impressive success of BCDcT, especially in hematological malignancies, its initial use was limited to last-resort cases due to the high morbidity associated with cytokine release syndrome (CRS) and immune cell-associated neurological toxicity (ICANS). Recent improvements in the safety profile of BCDcTs and better clinical management have lowered the incidences of CRS and ICANS, leading to its earlier use in some cancers. The success of BCDcT in B cell malignancies has promoted strong interest in applying this therapeutic approach to autoimmune disorders, particularly AINMD. This review will cover cell therapy fundamentals, limitations of current BCDTs, experiences with cell therapy in AINMD, and both ongoing and planned trials in this field.
2026-03-13 | CAR T cell therapy in autoantibody-mediated neurological disorders: a promising strategy.
Chimeric antigen receptor (CAR) T cell therapy is emerging as a promising approach for B cell-driven neurological autoimmune disorders, particularly those characterized by pathogenic autoantibodies that target key neural structures. These conditions, including neuromyelitis optica spectrum disorder, myasthenia gravis, Lambert-Eaton myasthenic syndrome, MOG antibody-associated disease, anti-NMDA receptor encephalitis, Diacylglycerol lipase alpha antibody associated encephalitis, stiff person syndrome, and multiple sclerosis, can be categorized based on their primary autoantigens into (1) extracellular antigen-associated (e.g., AQP4, AChR, NMDAR, MOG, VGCC), (2) intracellular antigen-associated (e.g., GAD65, DAGLA), or (3) unidentified antigenic origin (as seen in multiple sclerosis). This distinction is essential for guiding therapeutic strategies and exploring novel principles represented in distinct treatment approaches and their corresponding therapeutic outcomes. In this review, we propose a classification of CAR T cell therapies designed for different target antigens, including: CD19/20/BCMA-directed CAR T cells targeting general B cell-mediated pathogenesis, regulatory T cells modified with CARs, and the design of chimeric autoantibody receptors (CAARs) to selectively deplete pathogenic B cells directly associated with disease progression while preserving immune tolerance. We further discuss preclinical and clinical advancements, key challenges such as safety concerns and neurotoxicity, and the future landscape of CAR T applications in neuromyelitis optica spectrum disorder, myasthenia gravis, Lambert-Eaton myasthenic syndrome, MOG antibody-associated disease, anti-NMDA receptor encephalitis, Diacylglycerol lipase alpha antibody associated encephalitis, stiff person syndrome, and multiple sclerosis according to the latest research, case and trial data. CAR T cell therapy potentially offers a highly specific and effective method with thorough elimination of autoreactive B cells, representing a rapidly evolving field with the potential to transform the treatment of autoimmune neurological disorders. As CAR T technology advances, it holds the potential to become a groundbreaking immunoablative strategy with so-far disclosed controllable side effects; however, further long-term follow-up data are still needed to validate its application in autoimmune disorders.
2025-06-27 | A Rare Case of Lambert-Eaton Myasthenia Syndrome With Dysphasia and Dysarthria.
Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder resulting in muscle weakness. LEMS typically presents with progressive proximal muscle weakness, especially in the lower extremities, areflexia, and autonomic dysfunction. Bulbar involvement and respiratory compromise tend to occur later in the disease course and usually in severe, advanced cases. We report a unique case of a 50-year-old male with a known history of LEMS, autoimmune retinopathy, erectile dysfunction, and prediabetes who presented with progressive bulbar symptoms - slurred speech and difficulty swallowing - without accompanying weakness in the upper or lower extremities. Despite receiving four doses of intravenous immunoglobulin (IVIG) prior to admission with no improvement, he showed a significant clinical response to six sessions of plasmapheresis. Notably, the absence of limb weakness and preserved motor strength throughout his hospital stay raised diagnostic uncertainty, prompting evaluation for myasthenia gravis, which was ruled out through negative acetylcholine receptor antibody testing and reaffirmed voltage-gated calcium channel (VGCC) antibody positivity. Chest imaging showed no underlying malignancy. This case illustrates an atypical LEMS presentation dominated by severe bulbar dysfunction in the absence of limb involvement or respiratory failure, which is uncommon and may mimic other neuromuscular junction disorders such as myasthenia gravis. The favorable response to plasmapheresis emphasizes the importance of early recognition and aggressive immunomodulatory treatment in preventing clinical deterioration. Clinicians should maintain a high index of suspicion for LEMS even in unusual presentations to ensure timely and appropriate management.
2024-10-25 | Anti-CD19 CAR-T cells are effective in severe idiopathic Lambert-Eaton myasthenic syndrome.
Lambert-Eaton myasthenic syndrome (LEMS) is an autoantibody-mediated disease of the neuromuscular junction characterized by muscular weakness. Autoantibodies to presynaptic P/Q-type voltage-gated calcium channels (VGCCs) induce defective neuromuscular function. In severe cases, current immunosuppressive and immunomodulatory treatment strategies are often insufficient. First reports show beneficial effects of anti-CD19 chimeric antigen receptor (CAR)-T cell therapy in patients with autoantibody-mediated myasthenia gravis. We report a patient with isolated idiopathic LEMS treated with autologous anti-CD19-CAR-T cells. In this patient, CAR-T infusion leads to expansion of predominantly CD4+ CAR-T cells with a terminally differentiated effector memory cells re-expressing CD45RA (TEMRA)-like phenotype indicating cytotoxic capabilities and subsequent B cell depletion. VGCC antibody titers decrease, resulting in a clinical improvement of LEMS symptoms, e.g., 8-fold increase in walking distance. The patient does not show relevant side effects except for cytokine release syndrome grade 2 and intermittent neutropenia suggesting that anti-CD19 CAR-T cell therapy may be a treatment option in patients with LEMS.
2024-06-06 | Treatment of concomitant myasthenia gravis and Lambert-Eaton myasthenic syndrome with autologous CD19-targeted CAR T cells.
Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR T cells for treating concomitant MG/LEMS. After CAR T cell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage-gated calcium channel N-type autoantibody levels paralleled major neurological responses. Within 2 months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4 months post-CAR T cell infusion, respectively. This report highlights the potential for anti-CD19 CAR T cells to achieve profound clinical effects in the treatment of neuroimmunological diseases.
antibodies
2026-01-24 | Clinical Spectrum and Outcomes of SOX1 Antibody-Associated Paraneoplastic Neurological Syndromes: A Chinese Cohort Study.
SOX1 antibody-positive paraneoplastic neurological syndromes (PNS) exhibit significant population-specific clinical heterogeneity. While Western cohorts predominantly manifest Lambert-Eaton myasthenic syndrome (65%-80%), comprehensive clinical characterization and treatment response data in Asian populations remain critically limited. We conducted a single-center retrospective case series analyzing 13 consecutive patients with SOX1 antibody-positive PNS treated at Guangdong Sanjiu Brain Hospital from January 2019 to December 2024. SOX1 antibodies were confirmed using commercial immunoblot assay. Primary endpoints included treatment response (≥ 1-point improvement on modified Rankin Scale [mRS]) and functional recovery (mRS ≤ 2). Statistical analyses employed Fisher's exact tests and Mann-Whitney U tests. Among 13 patients (median age 61 years [IQR 56-67], 53.8% female), neuropsychiatric presentations predominated, including seizures (46.2%) and psychiatric symptoms (30.8%), with combined neuropsychiatric manifestations occurring in 53.8% of patients. Co-existing neuronal antibodies were identified in 15.4% of cases (GABAB receptor, LGI1). Malignancy was confirmed in 30.8% of patients. Immunotherapy recipients (n = 7) demonstrated significantly superior functional outcomes compared to non-treated patients: median 3-month mRS 0 (IQR 0-0) versus 3 (IQR 3-3), p = 0.03. Treatment response rates were 85.7% versus 33.3% (p = 0.103). Chinese patients with SOX1 antibody-positive PNS demonstrate a neuropsychiatric-predominant phenotype (53.8%), contrasting markedly with Western cohorts. Early immunotherapy administration was associated with superior functional outcomes (median 3-month mRS: 0 vs. 3, p = 0.03). These findings support comprehensive neuronal antibody profiling and early immunotherapy consideration in patients presenting with neuropsychiatric manifestations.
2026-01-08 | Coexisting cerebellar ataxia and Lambert-Eaton myasthenic syndrome without malignancy: insights from a case and systematic literature review.
BACKGROUND: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder often associated with small-cell lung carcinoma (SCLC). Cerebellar degeneration can coexist with LEMS, although their simultaneous presentation in the non-neoplastic setting is uncommon. METHODS: We describe a 75-year-old man with progressive cerebellar ataxia followed by bilateral ptosis, ophthalmoparesis, and proximal weakness. LEMS was confirmed by positive P/Q-type VGCC antibodies and electrophysiology. Extensive malignancy screening, including PET-CT, was negative, and immunotherapy produced marked neuromuscular and partial cerebellar improvement. To contextualize this case, we systematically reviewed the published literature (January 1991- August 2025) for this association, including cohorts, case reports and series providing individual patient-level details. RESULTS: We identified 37 studies reporting 67 patients with coexistent LEMS and cerebellar ataxia. Malignancy was present in 71.6% (48/67), predominantly SCLC (35/67, 52.2%). The median interval from neurological symptom onset to cancer diagnosis was 3 months (IQR 0.5–6.0; range − 60 to 39 months). Anti-VGCC antibodies were detected in 83.6% (56/67), anti-SOX1 in 7.5% (5/67), and anti-Hu in 3.0% (2/67), while 6.0% (4/67) were seronegative. Notably, 28.4% (19/67) had no malignancy during follow-up, suggesting a non-paraneoplastic autoimmune subset. Neurological improvement was comparable between paraneoplastic and non-paraneoplastic cases, but mortality was significantly higher in malignancy-associated cases (27.1% vs. 5.3%, p = 0.04). CONCLUSION: Coexistent LEMS and cerebellar ataxia is not invariably paraneoplastic. Early immunotherapy may improve neurological outcomes even in cancer-negative cases, but long-term oncologic surveillance remains essential.
2025-12-03 | Case Report: A Patient with Lambert-Eaton Myasthenic Syndrome Successfully Treated with Efgartigimod.
Lambert-Eaton myasthenic syndrome (LEMS) is a neuromuscular junction disorder which is frequently associated with tumors. Those patients generate pathogenic antibodies such as voltage-gated calcium channel (VGCC) antibodies. Existing studies have established the efficacy of efgartigimod in MG. This article reports the effect of efgartigimod following its administration to a patient with LEMS. A 73-year-old male was diagnosed with small cell lung cancer two years ago. Gradually he developed difficulties in speaking, eating, and lifting his head, along with limb weakness. The neuroelectrophysiological examination findings were consistent with LEMS. Laboratory tests detected VGCC antibodies and SOX-1 antibodies in the serum. The patient's symptoms continued to worsen after one week of corticosteroids use. Consequently, efgartigimod at a dose of 10 mg/kg was added once a week. After three weeks of treatment, the patient's clinical manifestations had improved significantly. Laboratory tests revealed a decrease in serum VGCC antibody and IgG levels. This is the first report on the use of efgartigimod in LEMS. The patient demonstrated substantial clinical improvement following treatment. Nevertheless, more clinical studies are required to verify whether efgartigimod offers a novel treatment approach for patients with LEMS.
2025-11-26 | Lambert-Eaton myasthenic syndrome presenting with occult mediastinal small cell carcinoma and positivity for anti-CV2/CRMP5 and anti-SOX1 antibodies: a case report.
BACKGROUND: Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune neuromuscular disorder characterized by proximal muscle weakness, autonomic dysfunction and hyporeflexia. Numerous studies have reported the association of LEMS with anti-SOX1 antibodies and small cell carcinoma, but LEMS with double antibody positivity and cerebellar ataxia accompanied by small cell carcinoma is relatively rare. CASE PRESENTATION: A 56-year-old female patient developed progressive limb weakness in November 2023, with symptoms gradually worsening over six months to include gait instability, impaired grip, chest tightness, dyspnea, dysphagia, and aspiration. Electrodiagnostic testing confirmed Lambert-Eaton myasthenic syndrome (LEMS) with positive VGCC antibodies. Paraneoplastic antibody testing showed positive anti-CV2/CRMP5 antibodies (24 AU) and borderline anti-SOX1 antibodies (9 AU), while other antibodies were negative. Initial imaging (contrast-enhanced chest CT and PET-CT) revealed no abnormalities, but follow-up one year later detected mediastinal small cell lung cancer. The patient underwent three plasma exchange sessions with corticosteroid therapy with limited response, then showed significant improvement after switching to subcutaneous Telitacicept (80 mg) combined with Fampyra Comprimidos and Mycophenolate Mofetil capsules, achieving independent ambulation and normal grip strength with only mild residual limb weakness. The patient has since been transferred to a local hospital for chemotherapy. CONCLUSION: We find that LEMS with concurrent anti-SOX1 and anti-CV2/CRMP5 positivity manifests as rapid progression, prominent proximal weakness, and potential cerebellar and autonomic involvement. Early and sustained oncological surveillance—particularly of mediastinal lymph nodes—and combined immunotherapeutic strategies can significantly improve neurological outcomes.
2025-01-22 | Association of Lambert-Eaton Myasthenic Syndrome and First Episode Psychosis: A Case Report.
Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune neuromuscular junction disorder characterized by proximal weakness, autonomic dysfunction, and areflexia associated with antibodies against voltage-gated calcium channels (VGCCs). Psychotic symptoms can occur in many autoimmune neurological disorders but they have rarely been observed in myasthenic syndromes. We report the case of a 21-year-old woman with primary autoimmune LEMS due to anti-VGCC antibodies subtype P/Q, who developed psychotic symptoms 3 years after the onset of motor symptoms. These symptoms decreased after every cycle of monthly intravenous immunoglobulin therapy. Different causes of reversible psychosis were excluded, such as autoimmune encephalitis. Due to a worsening of the patient's muscle strength, and the psychotic episodes, the patient received several treatments including an admission to a neurology unit. To our knowledge, this is the first described case of psychotic symptoms associated with LEMS. We speculate that VGCC antibodies could have a role in the development of mental symptoms, although additional hypotheses are also discussed.
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Drug Discovery Landscape
5 orphan drug designations for Lambert-Eaton myasthenic syndrome, including 1 approved therapy.
5 orphan drug designations for Lambert-Eaton myasthenic syndrome, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
3,4 diaminopyridine and choline bitartrate | small molecules | FDA | 2010-04-30 | — | MS Therapeutics Limited |
amifampridine [Firdapse] | small molecules | FDA | 2009-11-12 | 2022-09-29 | Catalyst Pharmaceuticals, Inc. |
Amifampridine phosphate [Firdapse] | small molecules | EMA | 2002-12-18 | — | Biomarin International Limited |
amifampridine | small molecules | FDA | 1990-12-18 | — | Catalyst Pharmaceuticals, Inc. |
Dynamine | small molecules | FDA | 1990-02-05 | — | Mayo Foundation |
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