Myasthenia Gravis
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  • ||||||||||  Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Navigating Generalized Myasthenia Gravis: Unraveling the Long-Term Efficacy and Convenience of Ravulizumab (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_578;    
    This case series suggests Ravulizumab as a potential chronic treatment for AChR ab-positive MG, with extended 7-8-week convenient infusion intervals, symptom-free intervals, stabilizing symptoms, and improving MG-ADL scores. We noted decreased exacerbations and no significant adverse effects; however, continuous monitoring is essential.
  • ||||||||||  Navigating the diagnosis journey: Depression and Myasthenia Gravis (Virtual) -  Feb 16, 2024 - Abstract #MDA2024MDA_575;    
    The proportion experiencing depression during this early-phase was higher than the US GP average, but lower than previous MG estimates. Understanding the depression trajectory, potential triggers, and impacts can inform health care providers and caregivers to provide early, interventional mental health support.
  • ||||||||||  ClC-1 inhibitor compound enhances skeletal muscle function in pre-clinical animal models of neuromuscular dysfunction (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_462;    
    Specifically, we found that compound muscle actions potentials and stimulated muscle force were both markedly improved when animals were dosed with a ClC-1 inhibitor. These findings suggest ClC-1 inhibition as a novel approach to enhancing neuromuscular transmission, thereby leading to improved muscle function and restored mobility, in disorders where neuromuscular transmission is compromised.
  • ||||||||||  Partnering with Patients and Care Partners to Guide the Design of a Generalized Myasthenia Gravis Real World Study (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_457;    
    Involving patients in the design of research is crucial in understanding how to ensure outcomes are patient-centric with a reasonable study burden. Insights from this innovative research design directly impacted the development of a large, global RW study, thus supporting recruitment goals, adherence to the study protocol, and the overall success and relevance of this research to patients with gMG.
  • ||||||||||  Incidence and Prevalence of Myasthenia Gravis: Analysis of a US Commercial Insurance Claims Database (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_456;    
    MG identification relied on billing diagnoses and was limited primarily to Medicaid and commercially insured patients, therefore older patients are likely underrepresented. However, the validity of the datasets used in this analysis give particular strength to the prevalence, incidence, and demographics observed in this study, which support the need for continued research in MG.
  • ||||||||||  Looking into the Future of Myasthenia Gravis Management Using Artificial Intelligence (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_446;    
    To mitigate myasthenic crisis risk, accessible tools for tracking symptoms are vital for patients and providers. The emergence of AI-driven models for automated symptom assessment holds promise for precise, patient-focused tools to enhance MG management in the future.
  • ||||||||||  Patient Preferences for Generalized Myasthenia Gravis Treatment Profiles: Results of a Web-Based Survey (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_444;    
    The first BWS exercise assessed preferences across 4 different treatment profiles (eg, similar to: ravulizumab, eculizumab, efgartigimod intravenous and subcutaneous, or zilucoplan). Patients with gMG preferred treatments with less frequent dosing schedules and consistent disease control, and consistent disease control was considered the most important attribute when choosing a therapy.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    The Patient Experience in Myasthenia Gravis: A Survey of 109 Individuals Regarding Their Care Journey (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_442;    
    Many patients experienced diagnostic delays and therapeutic side effects that lead to discontinuation or impeded daily function. Improvements in clinical perceptions/communication, access to specialized care, and expanded therapies are needed to improve quality-of-life in this population.
  • ||||||||||  Using Speech Biomarkers to Identify and Track Neuromuscular and Neurodegenerative Disorders (Poster Hall) -  Feb 16, 2024 - Abstract #MDA2024MDA_384;    
    Machine learning models demonstrated strong capabilities in predicting clinical diagnoses and outcomes with high accuracy and sensitivity. Conclusion Our findings highlight the potential of BioDigit Speech as a valuable tool to aid in identifying and tracking multiple neuromuscular and neurodegenerative disorders.
  • ||||||||||  Trial primary completion date, Real-world evidence, Real-world:  Descriptive Analysis of Real-world Data Collected With ME&MGopen (clinicaltrials.gov) -  Feb 16, 2024   
    P=N/A,  N=500, Recruiting, 
    Conclusion Our findings highlight the potential of BioDigit Speech as a valuable tool to aid in identifying and tracking multiple neuromuscular and neurodegenerative disorders. Trial primary completion date: Feb 2024 --> Feb 2025
  • ||||||||||  Trial completion date, Trial primary completion date:  Acupuncture in Myasthenia Gravis (AcuMG) (clinicaltrials.gov) -  Feb 15, 2024   
    P=N/A,  N=20, Active, not recruiting, 
    Trial completion date: Jan 2025 --> Sep 2025 Trial completion date: Dec 2023 --> Mar 2024 | Trial primary completion date: Dec 2023 --> Mar 2024
  • ||||||||||  Biomarker, Trial completion date, Trial primary completion date:  Predictors and Prognostic Factors of Myasthenia Gravis Outcome (clinicaltrials.gov) -  Feb 15, 2024   
    P=N/A,  N=30, Recruiting, 
    Trial completion date: Dec 2023 --> Mar 2024 | Trial primary completion date: Dec 2023 --> Mar 2024 Trial completion date: Sep 2023 --> Aug 2024 | Trial primary completion date: Jul 2023 --> Jun 2024
  • ||||||||||  Descartes-08 / Cartesian Therap
    Trial completion date, Trial primary completion date, CAR T-Cell Therapy:  Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG) (clinicaltrials.gov) -  Feb 15, 2024   
    P2,  N=30, Recruiting, 
    Trial completion date: Sep 2023 --> Aug 2024 | Trial primary completion date: Jul 2023 --> Jun 2024 Trial completion date: Dec 2023 --> Mar 2026 | Trial primary completion date: Dec 2023 --> Mar 2025
  • ||||||||||  Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
    Enrollment change:  Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) -  Feb 15, 2024   
    P2,  N=53, Active, not recruiting, 
    Trial completion date: Dec 2023 --> Mar 2026 | Trial primary completion date: Dec 2023 --> Mar 2025 N=80 --> 53
  • ||||||||||  New trial:  MuSK 1000: MuSK Myasthenia 1000 Study (clinicaltrials.gov) -  Feb 15, 2024   
    P=N/A,  N=1000, Recruiting, 
  • ||||||||||  KYV-101 / Kyverna
    FULLY HUMAN CD19-TARGETED CAR T CELLS IN A CASE OF REFRACTORY ANTI-ACHR ANTIBODY POSITIVE MYASTHENIA GRAVIS (CARRON) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1572;    
    Following lymphodepletion with fludarabine and cyclophosphamide, the patient received a single infusion of 1x108 anti-CD19 CAR T cells (day 0)... Our case report may serve as a foundation for conducting clinical trials with CD19 CAR T cells in MG and/or other neuroimmunological disorders and we plan to present a 1-year data update.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Neonatal Fc receptor (FcRn)-A Novel Therapeutic Approach for Autoimmune Disease (Pubmed Central) -  Feb 14, 2024   
    As one example, the administration of an FcRn inhibitor, efgartigimod, reduced IgG and anti-acetylcholine receptor antibody levels in patients with generalized myasthenia gravis (gMG), and improved Myasthenia Gravis Activities of Daily Living score in the phase III trial...Since FcRn inhibitors have just begun to be used in clinical practice, it is important to accumulate real-world data regarding their efficacy and safety. (Received August 21, 2023; Accepted October 6, 2023; Published February 1, 2024).
  • ||||||||||  Journal, Causal relationship:  Causal relationship between gut microbiota and myasthenia gravis: a two-sample Mendelian randomization study. (Pubmed Central) -  Feb 9, 2024   
    The MR investigation has substantiated the suggestive causal connection between gut microbiota and MG, which may provide helpful insights for innovative therapeutic and preventative approaches for MG. Further randomized controlled trials are needed to elucidate the gut microbiota's precise role and therapeutic potential in the pathogenesis of MG.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD), Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Journal:  Novel uses of complement inhibitors in myasthenia gravis-Two case reports. (Pubmed Central) -  Feb 9, 2024   
    The effect of complement inhibition on cancer outcomes of ICI therapy is unknown. A possible biologic basis for complement inhibitors in reducing irAEs of ICI, especially in the presence of underlying autoimmune disease, merits evaluation.