Myasthenia Gravis
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  • ||||||||||  Direct Health Care Costs Associated With Myasthenia Gravis (MG) in Hungary: A Claims Database Analysis () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_1616;    
    The present research was designed to address a gap in the current understanding of the direct cost implications of the GM market in Hungary. The analysis can serve as an important starting point for the planning of the therapeutic area, as the introduction of new types of pharmaceutical products will result in a paradigm shift not only in terms of therapeutic efficacy but also in terms of the financial resources required.
  • ||||||||||  Healthcare Resource Consumption and Costs of Myasthenia Gravis in France () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_1272;    
    US adults self-report ing a physician diagnosis of AChR -Ab+ gMG completed a web-based , best MG-associated costs in France are high, especially in the first year of the disease and are mainly driven by hospitalizations.
  • ||||||||||  Incidence of Rare Diseases in a City From the Colombian Caribbean () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_943;    
    Rare diseases can be chronic or incurable. Its low incidence, compared to other conditions, in a city of less than 2 million inhabitants, like Barranquilla, motivates a detailed and detailed study of each one.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    Journal, Real-world evidence, Real-world:  Eculizumab in refractory myasthenia gravis: a real-world single-center experience. (Pubmed Central) -  Nov 4, 2024   
    This single-center real-world study confirmes safety and effectiveness of eculizumab. Eculizumab improved rapidlyall clinical outcome measures, leading to discontinuation of intravenous immunoglobulin treatment and remarkable immunosuppressant-sparing benefits.
  • ||||||||||  Journal:  Congenital myasthenic syndromes in adults: clinical features, diagnosis and long-term prognosis. (Pubmed Central) -  Nov 3, 2024   
    In conclusion, even if motor and/or respiratory deterioration could occur in patients with initially moderate disease, particularly in DOK7, SCCMS and GFPT1 patients, the long-term prognosis for most CMS patients was favourable, with neither ventilation nor wheelchair needed at last visit. CHRNE patients did not worsen during adulthood and RAPSN patients, often severely affected in early childhood, subsequently improved.
  • ||||||||||  Review, Journal:  New treatment strategies in Myasthenia gravis. (Pubmed Central) -  Oct 31, 2024   
    The integration of new molecular therapies into clinical practice could significantly transform the treatment landscape of MG, offering more effective and tailored therapeutic options for patients who do not respond adequately to traditional treatments. These innovations underscore the importance of ongoing research and clinical trials to optimize therapeutic strategies and improve the quality of life for individuals with MG.
  • ||||||||||  Review, Journal:  Strategies to improve autoimmune neurological diseases treatment. (Pubmed Central) -  Oct 31, 2024   
    It also discusses the role of B- and T-cells in autoimmune neurology and how these can be exploited therapeutically. Finally, it argues for improved training of present and future neuroimmunologists.
  • ||||||||||  remdesivir / Generic mfg., methylprednisolone sodium succinate / Generic mfg.
    Journal:  Complications of Long COVID: Unraveling a Case of Very-Late-Onset Myasthenia Gravis. (Pubmed Central) -  Oct 31, 2024   
    Of note, approximately four months prior to this admission, he was diagnosed with COVID-19 pneumonia and was treated appropriately with remdesivir and corticosteroids...The patient received treatment consisting of pyridostigmine, a pulse dose of methylprednisolone, and intravenous immunoglobulin (IVIG) therapy...The pathophysiology linking COVID-19 to MG primarily involves immune dysregulation triggered by the viral infection, which may disrupt immune tolerance and lead to clinical autoimmunity. This case stresses the need for vigilance in diagnosis and managing neurological complications in the context of viral respiratory illnesses, particularly in vulnerable populations.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Efgartigimod treatment for generalized myasthenia gravis: a single-center case series of 16 patients. (Pubmed Central) -  Oct 29, 2024   
    Improvements in the outcomes of patients with gMG, including patients with severe gMG, myasthenic crisis, and refractory to anti-complementary therapy, were observed after the first cycle of efgartigimod treatment. Our real-world experience in Japan suggests the future possibilities for the treatment with efgartigimod for gMG with diverse backgrounds.
  • ||||||||||  Journal, Surgery:  Impact of the expanded indication of robot-assisted thoracic surgery for mediastinal tumors. (Pubmed Central) -  Oct 29, 2024   
    RATS has recently been expanded for mediastinal tumors with improved short-term outcomes in our hospital after health insurance was applied in Japan. In the future, it will be necessary to discuss the further expansion of its indications by taking into account safety and long-term outcomes.
  • ||||||||||  Review, Journal:  Targeted Treatments for Myasthenia Gravis in Children and Adolescents. (Pubmed Central) -  Oct 28, 2024   
    Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis.
  • ||||||||||  Opdivo (nivolumab) / BMS, Yervoy (ipilimumab) / BMS
    Journal, PD(L)-1 Biomarker, IO biomarker, Metastases:  Management of overlapping immune-related myocarditis, myositis, and myasthenia in a young patient with advanced NSCLC: a case report. (Pubmed Central) -  Oct 28, 2024   
    A 40-year-old patient, diagnosed with stage IV non-oncogene addicted lung adenocarcinoma, with nivolumab-ipilimumab-chemotherapy as first-line treatment, developed a rare myocarditis-myositis-myasthenia gravis overlap syndrome...Treatment with oral prednisone, pyridostigmine, and IV Igs was started due to poor clinical response followed by methylprednisolone...This report aims to raise awareness among physicians about these serious side effects. A multidisciplinary approach led to clinical improvement and early intervention, optimizing patient outcomes.
  • ||||||||||  Review, Journal:  Application of novel CAR technologies to improve treatment of autoimmune disease. (Pubmed Central) -  Oct 24, 2024   
    These include logic-gated CARs, soluble protein-secreting CARs, and modular CARs that enable CAR T cell therapies to be more specific, reach a wider span of target cells, be safer for patients, and give a more potent cytotoxic response. Applying these novel CAR technologies to the treatment of autoimmune diseases has the potential to revolutionize this growing application of CAR T cell therapies.