Myasthenia Gravis
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  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Lunch 'n Learn: New Frontiers in the management of myasthenia gravis (H-M2: Alberta) -  Jun 12, 2023 - Abstract #CNSF2023CNSF_131;    
    It is an unaccredited learning activity and not eligible for MOC credits. Learning Objectives: Understand the evolving treatment landscape for patients with myasthenia gravis Understand the role of IgG autoantibodies in the pathophysiology of myasthenia gravis Understand the mechanism of action of efgartigimod Understand the medical evidence for efgartigimod in the treatment of patients with myasthenia gravis Learning Objectives: Understand the evolving treatment landscape for patients with myasthenia gravis Understand the role of IgG autoantibodies in the pathophysiology of myasthenia gravis Understand the mechanism of action of efgartigimod Understand the medical evidence for efgartigimod in the treatment of patients with myasthenia gravis
  • ||||||||||  Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
    Lunch 'n Learn: New Advances in the Treatment of General Myasthenia Gravis: Expanding the use of Targeted Therapy (H-M2: Alhambra) -  Jun 12, 2023 - Abstract #CNSF2023CNSF_52;    
    It is an unaccredited learning activity and not eligible for MOC credits. Learning Objectives: Define gMG management goals and current strategies Define the role of complement in the pathophysiology of gMG Evaluate the Efficacy and Safety of Ravulizumab in the treatment of (AchR) anti-body positive generalized Myasthenia Gravis Understand how the clinical evidence can be applied in the clinical setting through case discussion Learning Objectives: Define gMG management goals and current strategies Define the role of complement in the pathophysiology of gMG Evaluate the Efficacy and Safety of Ravulizumab in the treatment of (AchR) anti-body positive generalized Myasthenia Gravis Understand how the clinical evidence can be applied in the clinical setting through case discussion
  • ||||||||||  Advances in treatments of hereditary neuromuscular diseases in children and adults (CC: Theatre - 1st Flr) -  Jun 12, 2023 - Abstract #CNSF2023CNSF_3;    
    Gerald Pfeffer) and myasthenic syndromes (Dr. Hanns Lochmuller) Learning Objectives: Understand all available treatment options for children and adults with neuromusuclar disease Understand potential adverse events of treatments to optimize choice of therapy and monitoring Recognize the utility of magnetic resonance imaging and other diagnostic tests evaluating a patient with a neuromuscular disorder Learning Objectives: Understand all available treatment options for children and adults with neuromusuclar disease Understand potential adverse events of treatments to optimize choice of therapy and monitoring Recognize the utility of magnetic resonance imaging and other diagnostic tests evaluating a patient with a neuromuscular disorder
  • ||||||||||  OUTCOMES OF THERAPEUTIC PLASMA EXCHANGE: A ONE-YEAR EXPERIENCE IN INTERNAL MEDICINE INTENSIVE CARE UNIT (ePoster (Virtual Only)) -  Jun 9, 2023 - Abstract #ERAEDTA2023ERA_EDTA_2490;    
    However, it showed no significant difference(p?=?0.2754), although the EBV infection rate was higher in the thymomas with myasthenia gravis. According to our experience, membrane therapeutic plasma exchange is a successful and secure treatment for a variety of illnesses including thrombotic microangiopathies, systemic vasculitis, myasthenia gravis, proliferative lupus nephritis, and Guillain-Barr
  • ||||||||||  Retrospective data, Journal, Metastases:  Outcomes of extended resection for locally advanced thymic malignancies. (Pubmed Central) -  Jun 9, 2023   
    In an expert center, extended resection targeting complete resection rather than organ preservation provided good outcomes in patients with locally advanced thymic malignancies. The risk/benefit ratio of surgery should be assessed with special care in patients who are elderly or have myasthenia gravis.
  • ||||||||||  Rystiggo (rozanolixizumab) / UCB
    Trial completion date, Trial primary completion date:  A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Jun 7, 2023   
    P3,  N=165, Active, not recruiting, 
    These results may provide a potential clue for doctors, nurses, and other caregivers to optimise treatment strategies for targeted patients with MG. Trial completion date: Oct 2023 --> Jan 2024 | Trial primary completion date: Oct 2023 --> Jan 2024
  • ||||||||||  prednisone / Generic mfg.
    Retrospective data, Journal:  Clinical and immune-related factors associated with exacerbation in adults with well-controlled generalized myasthenia gravis. (Pubmed Central) -  Jun 6, 2023   
    Multivariable Cox regression analysis showed that age at onset, disease duration before enrollment, Myasthenia Gravis Foundation of America classification (MGFA) class III vs. class II, MGFA class IV-V vs. class II, AChR-ab levels, anti-muscle specific kinase antibody levels, thymus hyperplasia, prednisone plus immunosuppressants vs. prednisone treatment, and thymectomy were independent predictors for exacerbations [hazard ratio (HR) = 1.011, 1.031, 1.580, 1.429, 2.007, 2.033, 1.461, 0.798, and 0.651, respectively]...Myasthenia gravis (MG) exacerbations were more frequent in those patients with older onset age, longer disease duration, more severe MGFA classification, positive AChR-ab, and lack of combined immunotherapy or thymectomy treatment. On the other hand, CD3CD19 B cells, CD3CD8 T cells, Tregs, and AChR-ab in peripheral blood may be involved in the course of GMG exacerbation.
  • ||||||||||  Journal:  Autoimmunity to Neuronal Nicotinic Acetylcholine Receptors. (Pubmed Central) -  Jun 5, 2023   
    Neuronal-type nAChRs are also present in several non-excitable tissues, however the presence and possible role of antibodies against them needs further verification. It is likely that the future development of more sensitive and disease-specific assays would reveal that neuronal nAChR autoantibodies are much more frequent and may explain the mechanisms of some seronegative autoimmune diseases.
  • ||||||||||  Retrospective data, Journal:  The Value of Postoperative Radiotherapy in Thymoma Patients with Myasthenia Gravis. (Pubmed Central) -  Jun 5, 2023   
    It is likely that the future development of more sensitive and disease-specific assays would reveal that neuronal nAChR autoantibodies are much more frequent and may explain the mechanisms of some seronegative autoimmune diseases. Overall, our findings indicate that PORT positively impacts thymoma patients with MG, particularly those with a higher histologic subtype and Masaoka-Koga staging.
  • ||||||||||  Review, Journal:  Review of Neurological Manifestations of SARS-CoV-2. (Pubmed Central) -  May 31, 2023   
    The systemic inflammatory mediators likely activate astrocytes and microglia across the blood-brain barrier, indirectly affecting CNS-specific immune activation and tissue injury. The management differs according to co-morbidities and the neurological disorder.
  • ||||||||||  Review, Journal:  Neuromuscular Disorders Associated With COVID-19. (Pubmed Central) -  May 31, 2023   
    Complications have been documented both as a direct result of primary infection but also in those with pre-existing neuromuscular disorders from myasthenia gravis to devastating critical illness neuromyopathies. In this review, we will discuss the relationship between COVID-19 infection and critical illness neuromyopathy, peripheral nerve palsies, myalgias, positional compressive neuropathy, myasthenia gravis, and Guillain-Barr
  • ||||||||||  Review, Journal:  FOXM1: Functional Roles of FOXM1 in Non-Malignant Diseases. (Pubmed Central) -  May 31, 2023   
    The complex mechanisms involve multiple signaling pathways, such as WNT/?-catenin, STAT3/FOXM1/GLUT1, c-Myc/FOXM1, FOXM1/SIRT4/NF-?B, and FOXM1/SEMA3C/NRP2/Hedgehog. This paper reviews the key roles and functions of FOXM1 in kidney, vascular, lung, brain, bone, heart, skin, and blood vessel diseases to elucidate the role of FOXM1 in the development and progression of human non-malignant diseases and makes suggestions for further research.
  • ||||||||||  Trial completion date, Trial primary completion date, Real-world evidence, Real-world:  The Impact of Myasthenia Gravis in the Real World (clinicaltrials.gov) -  May 30, 2023   
    P=N/A,  N=2000, Recruiting, 
    This paper reviews the key roles and functions of FOXM1 in kidney, vascular, lung, brain, bone, heart, skin, and blood vessel diseases to elucidate the role of FOXM1 in the development and progression of human non-malignant diseases and makes suggestions for further research. Trial completion date: Dec 2022 --> May 2024 | Trial primary completion date: Dec 2022 --> May 2024
  • ||||||||||  verapamil / Generic mfg., dantrolene / Generic mfg.
    Episodic exercise-induced gait disorder in a child (Foyer 3B, 3rd Floor) -  May 29, 2023 - Abstract #EPNS2023EPNS_799;    
    Pathogenic mutations in ATP2A1 gene cause Brody disease which is a rare, autosomal recessive myopathy leading to exercise-induced muscle stiffness. that may worsen upon exposure to cold temperatures.There is an increased risk of Rhabdomyolysis and malignant hyperthermia .
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. (Pubmed Central) -  May 29, 2023   
    Furthermore, in an interim analysis of the ongoing open-label phase 3 ADAPT+ extension trial, efgartigimod alfa provided consistent clinically meaningful improvements in patients with gMG. Efgartigimod alfa was generally well tolerated, with most adverse events being mild to moderate in severity.
  • ||||||||||  Journal:  Greater Number of Plasma Exchanges Does Not Improve Outcome in Myasthenic Crisis. (Pubmed Central) -  May 27, 2023   
    Thymectomies are motivated more by symptoms than by medication, and a lack of neurologist discussion is the most common barrier to thymectomy. This study provides class IV evidence that extending the number of plasma exchanges beyond 5 does not correlate with decreased hospital length of stay or improved discharge disposition in patients experiencing myasthenic crisis.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Neonatal Fc Receptor Inhibitor Therapeutics in Neuromuscular Disease. (Pubmed Central) -  May 27, 2023   
    Recently, the FcRn inhibitor efgartigimod was approved for the treatment of myasthenia gravis...Other disorders traditionally treated with IVIg may also benefit from FcRn inhibition in certain contexts. This manuscript discusses the mechanism of FcRn inhibition, preclinical data, and the results of clinical trials of this agent for a wide range of neuromuscular diseases.
  • ||||||||||  Journal, PD(L)-1 Biomarker, IO biomarker:  Circulating CXCR5 natural killer cells are expanded in patients with myasthenia gravis. (Pubmed Central) -  May 24, 2023   
    Furthermore, CXCR5 NK cells inhibited plasmablast differentiation, while CXCR5 NK cells could more efficiently promote B cell proliferation. These results reveal that CXCR5 NK cells exhibit distinct phenotypes and functions compared with CXCR5 NK cells and might participate in the pathogenesis of MG.
  • ||||||||||  Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
    Review, Journal:  Ravulizumab: A Review in Generalised Myasthenia Gravis. (Pubmed Central) -  May 22, 2023   
    Efficacy and tolerability data for up to 1 year from the ongoing open-label extension phase are consistent with those from the randomized, placebo-controlled phase; further results are awaited with interest. Thus, ravulizumab is an efficacious, generally well tolerated and convenient treatment option in adults with AChR Ab+ gMG, expanding the options available for gMG management.
  • ||||||||||  Journal:  Conventional and emerging treatments and controversies in myasthenia gravis. (Pubmed Central) -  May 22, 2023   
    The mechanisms of action of new drugs and the immunopathogenesis of different MG subtypes must be considered in therapy decision making. Integrating new agents in the treatment scenario of MG can significantly improve disease management.
  • ||||||||||  levofloxacin / Generic mfg.
    Journal:  Levofloxacin as a possible cause of myasthenic crisis. (Pubmed Central) -  May 22, 2023   
    Integrating new agents in the treatment scenario of MG can significantly improve disease management. No abstract available
  • ||||||||||  Rituxan (rituximab) / Roche
    New P3 trial:  Rituximab EfFicacy IN MyasthEnia Gravis (REFINE) (clinicaltrials.gov) -  May 22, 2023   
    P3,  N=40, Recruiting, 
  • ||||||||||  Journal:  Therapeutic Strategies for Myasthenia Gravis (Pubmed Central) -  May 21, 2023   
    Molecular-targeted drugs for MG have recently been developed. To date, three such drugs are available in Japan.
  • ||||||||||  THE DISEASE GENOMIC GRAMMAR OF MYASTHENIA GRAVIS INDICATES A COMMON GENETIC BACKGROUND WITH RHEUMATOID ARTHRITIS () -  May 19, 2023 - Abstract #EULAR2023EULAR_4227;    
    Identifying the optimal genomic grammar in MG with the use of novel Bioinformatic tools may assist in the better understanding of this polygenic disease, while its association with RA and other ADs is a helpful step towards comprehending the shared biological pathways leading to autoimmunity. The data resulting from our study can be utilized towards the development of an application designed to assist clinical diagnosis by using the patients
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Safety Profile of Efgartigimod from Clinical Trials in Participants with Immunoglobulin G-Mediated Autoimmune Diseases (Room:Exhibition) -  May 18, 2023 - Abstract #ISTH2023ISTH_1963;    
    Conclusion This ongoing phase 2 study will evaluate the safety and efficacy of nipocalimab in adults with active SLE, using multiple clinical outcome measures. Across all indications and doses studied, efgartigimod demonstrated a consistent safety profile, with comparable treatment emergent adverse event (TEAE) rates to placebo (ADVANCE 93.0% efgartigimod vs. 95.6% placebo; ADAPT 77.4% efgartigimod vs. 84.3% placebo; 85% of participants in open label pemphigus study).