Myasthenia Gravis
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  • ||||||||||  Retrospective data, Journal:  Clinical Characteristics of Double Seropositive Myasthenia Gravis. (Pubmed Central) -  Oct 21, 2024   
    Nine patients were administered immunosuppressants, three patients were given intravenous immunoglobulin, and a single patient underwent plasmapheresis. Our study shows that DSP-MG is more similar to the clinical phenotype of AChR-MG.
  • ||||||||||  Journal:  Quantitative assessment of dysphagia in myasthenia gravis. (Pubmed Central) -  Oct 21, 2024   
    Among these parameters, the incidence of an excessive expiratory flow, cluster of excessive expiratory flows, and prolonged EMG pattern significantly improved after treatment (p <0.05). Conclusion Based on this study, the respiratory patterns and submental sEMG are likely to reflect the severity of pharyngeal muscle weakness/fatigability and thus can be used as a quantitative parameter for dysphagia in patients with MG.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Case report: Rapid symptom relief in autoimmune encephalitis with efgartigimod: a three-patient case series. (Pubmed Central) -  Oct 18, 2024   
    All three patients experienced marked and swift symptomatic relief after four cycles of efgartigimod treatment, with no complication. Current first-line and second-line treatments for AE have limitations, and efgartigimod has demonstrated potential in the rapid and efficacious treatment of AE, emerging as a promising option for the management of this disease.
  • ||||||||||  trimethoprim/sulfamethoxazole / Generic mfg.
    Review, Journal:  Myasthenia gravis complicated with pulmonary infection by Nocardia cyriacigeorgica: a case report and literature review. (Pubmed Central) -  Oct 17, 2024   
    The patient was prescribed trimethoprim-sulfamethoxazole...Additionally, we searched PubMed for case reports of Nocardia cyriacigeorgica pulmonary infection from 2010 to 2024 and conducted a statistical analysis of the case information. This report aims to highlights the increased risk of pulmonary Nocardia infection in MG patients after the use of steroids and immunosuppressants, thereby enhancing clinical awareness.
  • ||||||||||  Review, Journal:  CAR immunotherapy in autoimmune diseases: promises and challenges. (Pubmed Central) -  Oct 16, 2024   
    This report aims to highlights the increased risk of pulmonary Nocardia infection in MG patients after the use of steroids and immunosuppressants, thereby enhancing clinical awareness. This review examines the mechanisms, efficacy, and safety of CAR approaches with a focus on their use in autoimmune diseases including systemic lupus erythematosus, Sj
  • ||||||||||  Review, Journal:  Blood-Brain Barrier Disruption in Neuroimmunological Disease. (Pubmed Central) -  Oct 16, 2024   
    GRP78 autoantibodies were detected in paraneoplastic cerebellar degeneration and Lambert-Eaton myasthenic syndrome, and they were associated with cerebellar ataxia with anti-P/Q type voltage-gated calcium channel antibodies. This review reports that therapies affecting the BBB that are currently available for disease-modifying therapies for neuroimmunological diseases have the potential to prevent BBB damage.
  • ||||||||||  The Rare Disease Research Network for Myasthenia Gravis, MGNet (Riverside Theatre (Plenary Hall)) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_367;    
    MGNet investigators actively collaborate with industry partners BioSensics and Care Constitution. The presentation will highlight MGNet's scientific contributions to the field of myasthenia gravis, the administrative challenges of managing a multi-institutional and international organization, and the vision for MGNet's future development.
  • ||||||||||  Agents that Worsen/Reveal MG (Riverview Room 5) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_364;    
    This talk explores the variety of agents implicated in worsening myasthenic control and critically assesses which are likely to have a significant risk versus those that are confounded by the natural variable nature of the disease (and thus false association). Patients and clinicians alike benefit from a rigorous understanding of which agents' warnings to take seriously, such that the person with myasthenia can avoid agents that may be dangerous but also is not unnecessarily deprived of effective treatment options.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    LEMS Update (Riverview Room 5) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_362;    
    Amifampridine is the first choice symptomatic treatment, while also a beneficial effect of pyridostigmine has been reported...Anti-CD 20 antibody therapies or CAR-T cell therapies might be interesting options for patients that are more difficult to treat. Importantly, LEMS is one of the rare autoimmune disorders with a well-defined pathogenesis due to a well-known autoantibody, which facilitates detailed studies of the effect of new drugs.
  • ||||||||||  Handling Fatigue in MG (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_354;    
    Exclusion of other co-morbid causes of fatigue, such as psychiatric disease, sleep disorders and metabolic or systemic disorders, is important. Different therapeutic approaches to fatigue management will be discussed.
  • ||||||||||  FcRn Antagonists (M8) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_349;    
    Unlike the 'broad-spectrum' conventional immunosuppressants and biological agents that act upstream in MG immunology, FcRn antagonists may only theoretically be below par in terms of mitigating MG immunopathology but have proven clinically efficacious. Further research and direct clinical comparisons among agents will be necessary to unravel these complexities.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Zilbrysq (zilucoplan) / UCB
    Complement in Myasthenia Gravis (M1) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_337;    
    In addition to their role in innate response, anaphylatoxins regulate the adaptive response either directly or indirectly through B-cell and T-cell activation. The complexity of the complement system in an autoimmune disease underscores the necessity to understand the off targets in any complement directed therapeutic.
  • ||||||||||  The Role of the Thymus in Autoimmunity (M1) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_336;    
    Autoantibodies produced in thymoma-associated MG are against the AChR, but also muscle autoantigens such as titin, the ryanodine receptor and neurofilament protein. Understanding the key factors that drive loss of tolerance in thymic hyperplasia and thymoma will enable identification of targets for therapeutic development.
  • ||||||||||  prednisone / Generic mfg.
    Thymectomy for Myasthenia Gravis: Analyzing Usage Patterns and Dispelling Safety Concern (M8) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_332;    
    However, the landscape changed in 2016 when the MGTX Trial demonstrated that thymectomy combined with prednisone was superior to prednisone alone in acetylcholine receptor (AChR) antibody-positive MG...Given the slow adoption of thymectomy despite demonstrated efficacy, the neuromuscular community must develop systems to enhance its utilization. Addressing this issue is critical to improving outcomes for MG patients.
  • ||||||||||  prednisone / Generic mfg.
    Thymectomy in Myasthenia Gravis (M8) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_331;    
    Patients were randomized to receive either extended transsternal thymectomy plus prednisone or medical management with prednisone...The 2020 update indicates that thymectomy "may be offered" to patients with AChR antibody non-thymomatous OMG who do not respond to acetylcholinesterase inhibitors or immunosuppressive agents or who have contraindications or prefer not to take immunosuppressants. Though data regarding thymectomy for Juvenile MG are limited to retrospective case series, it seems to suggest that patients who underwent thymectomy may improve in disease severity and have rare post-operative complications.
  • ||||||||||  Rituxan (rituximab) / Roche
    Effects of Age and Sex (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_322;    
    Most of the steroid sparing agents are contraindicated in pregnancy and biological agents like Rituximab are to be avoided during the last trimester of pregnancy...In an era where personalized and precision medicine are gaining importance, age and sex are presently the foremost variables to be considered while deciding on investigations and management pathways. This will continue to remain so even after other factors like ethnicity, more advanced immunological and genetic profiling are also considered when deciding the treatment protocol.
  • ||||||||||  Juvenile MG (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_321;    
    While a 2021 report in the literature summarizing several decades of recognized cases in Canada and Europe identified only 1-2 cases per year in those countries, infantile botulism is serious, treatable and likely underdiagnosed worldwide. Tick paralysis (with over 70 species secreting a toxin causing paralysis) is another example of toxic NMJ dysfunction occurring worldwide in children.
  • ||||||||||  Biomarkers (Riverside Theatre (Plenary Hall)) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_316;    
    Moving forward, the validation and multi-center evaluation of these promising biomarkers are essential to establish their sensitivity, specificity, and clinical utility. Ultimately, validated biomarkers hold the potential to revolutionize MG management by facilitating early intervention and personalized treatment strategies.
  • ||||||||||  PL.04 - Plenary Session 4: Outcome Measures and Treatment Guidelines (Riverside Theatre (Plenary Hall)) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_313;    
    Finally, since 2012 formal treatment recommendations have been developed in several countries, with the first international recommendation published in 2016. This plenary session will provide a background and update on each of these topics.
  • ||||||||||  Rituxan (rituximab) / Roche
    Rituximab (M1) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_300;    
    In the era of biologics with rapidly emerging alternative high efficacy therapies, the role of RTX in AChR+ MG is undefined. The speaker will discuss its role in MG therapeutics personalised to the individual patient, and the current movement towards RTX initiation early in the MG disease course as a disease modifying therapy.
  • ||||||||||  Tacrolimus in Myasthenia Gravis (M1) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_299;    
    Long-term use of tacrolimus in the patients with myasthenia gravis seems to be safe. To confirm the efficacy and safety of tacrolimus, additional large-scale studies of high quality are needed.
  • ||||||||||  FcRN Antagonists in MG (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_63;    
  • ||||||||||  Session Introduction (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_60;    
  • ||||||||||  TC.05 - Teaching Course: Congenital Hypotonia (M7) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_41;    
    It then goes on to give information on the three main groups of neuromuscular diseases that present as hypotonia and weakness in infancy, congenital myopathies, congenital myasthenic syndromes, and SMA and congenital muscular dystrophies. As treatments become available (eg. gene transfer for SMA), it becomes increasingly important that a correct diagnosis is arrived at as promptly as possible.
  • ||||||||||  Inflammation in Muscle Biopsy - Myositis or Not (Riverview Room 4) -  Oct 15, 2024 - Abstract #ICNMD2024ICNMD_38;    
    In such cases, a trial of immunotherapy may be warranted. However, aggressive to treat such patients is a difficult decision one needs to make with the patient and family, weighing risks versus benefits.
  • ||||||||||  Rystiggo (rozanolixizumab-noli) / UCB
    Review, Journal:  Rozanolixizumab: A New Therapy in the Treatment of Myasthenia Gravis. (Pubmed Central) -  Oct 14, 2024   
    The approval of rozanolixizumab represents an advancement in therapy for generalized myasthenia gravis. The provision of individualized, targeted, and well-tolerated treatment is valuable for the patients whose myasthenia gravis is not well controlled and who are seeking a medication with a rapid onset of action to improve their symptoms and overall quality of life.