Myasthenia Gravis
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  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
    An Indirect Comparison of Efgartigimod Versus Rituximab for Generalized Myasthenia Gravis (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1780;    
    The ITC showed improved efficacy of Efgartigimod vs Rituximab. However, due to limited ESS in the MAIC and the strong assumptions underlying the Bucher’s adjusted comparison, these results should be interpreted with caution.
  • ||||||||||  Vitiligo in Italy through Administrative Healthcare Data (Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1764;    
    Vitiligo is a highly undetected and untreated disease. Although this study underestimated its prevalence, Italian administrative healthcare data catched the higher concern among women and quantified the burden on the INHS of patients with a diagnosis of vitiligo and treated with phototherapy.
  • ||||||||||  Challenges in Presenting Engagement Statistics in Real-World Digital Studies (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1591;    
    Missing data can be an issue across all RWE, particularly for patient-reported data; the data must be correctly interpreted to track participants’ retention and engagement, ensuring they continue to share high-quality data. The concepts of attrition and completion should be separated to better manage digital real-world studies.
  • ||||||||||  Tavneos (avacopan) / Kissei, Amgen, Ultomiris IV (ravulizumab IV) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    250: Poster Tour: Rare and Orphan Diseases (In-person) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_255;    
    Up to 90% received a MG diagnosis during hospitalisation. Download the Rare and Orphan Diseases Poster Tour Guide Packet Posters featured in this tour: CO15: A Matching-Adjusted Indirect Comparison of Efgartigimod Versus Ravulizumab for Generalized Myasthenia Gravis EE382: Cost-Utility Analysis of Avacopan for the Treatment of Anca-Associated Vasculitis (AAV) Patients in the UK EE274: Orphan Drug Pricing Comparisons in Low-, Middle- and High-Income Countries EE147: Estimating the Causal Effect of Early Use of Erythropoietic Stimulating Agents in Intermediate-1 to Low-Risk MDS Patients: An Application of the Longitudinal Targeted Maximum Likelihood Estimation HPR17: Changing Landscape of Orphan Drug Reimbursement - Evidence From EU-4 and England SA73: Addressing Unmet Needs of Patients With Neuronopathic Gaucher Disease Type 2 and Type 3: Creation of the GARDIAN Patient Registry
  • ||||||||||  Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) / argenx
    Enrollment open:  Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Sep 1, 2022   
    P2/3,  N=12, Recruiting, 
    Download the Rare and Orphan Diseases Poster Tour Guide Packet Posters featured in this tour: CO15: A Matching-Adjusted Indirect Comparison of Efgartigimod Versus Ravulizumab for Generalized Myasthenia Gravis EE382: Cost-Utility Analysis of Avacopan for the Treatment of Anca-Associated Vasculitis (AAV) Patients in the UK EE274: Orphan Drug Pricing Comparisons in Low-, Middle- and High-Income Countries EE147: Estimating the Causal Effect of Early Use of Erythropoietic Stimulating Agents in Intermediate-1 to Low-Risk MDS Patients: An Application of the Longitudinal Targeted Maximum Likelihood Estimation HPR17: Changing Landscape of Orphan Drug Reimbursement - Evidence From EU-4 and England SA73: Addressing Unmet Needs of Patients With Neuronopathic Gaucher Disease Type 2 and Type 3: Creation of the GARDIAN Patient Registry Not yet recruiting --> Recruiting
  • ||||||||||  Journal:  Transcript-Based Diagnosis and Expanded Phenotype of an Intronic Mutation in TPM3 Myopathy. (Pubmed Central) -  Aug 31, 2022   
    This study broadens the phenotypic spectrum of recessive TPM3 disease, highlighting tongue fasciculations and bilateral clubfoot, as well as possibly-related cerebral atrophy. It also shows the importance of a broad approach to genetic analysis and the utility of RNA-based studies, demonstrating efficacy of early genome and transcriptome queries in facilitating rapid and cost-effective diagnosis of congenital myopathies.
  • ||||||||||  Review, Journal, Adverse events:  Poor responses and adverse outcomes of myasthenia gravis after thymectomy: Predicting factors and immunological implications. (Pubmed Central) -  Aug 31, 2022   
    However, in addition to these variations among individuals, pathological remnants and the abnormal immunological milieu and responses potentially represent major mechanisms that underlie the detrimental neurological outcomes after thymectomy. We underscore these plausible risk factors and discuss the immunological implications therein, which may be conducive to better managing the indications for thymectomy, to avoiding modifiable risk factors of poor responses and adverse outcomes, and to developing post-thymectomy preventive and therapeutic strategies for MG.
  • ||||||||||  Journal:  Motor neuron, peripheral nerve, and neuromuscular junction disorders. (Pubmed Central) -  Aug 30, 2022   
    Myasthenic crisis is a clinical challenge and is associated with substantial morbidity including prolonged mechanical ventilation and 5%-12% mortality. Emergency room physicians and consultant pulmonologists and neurologists must know such respiratory risks, be able to recognize early signs, and treat properly.
  • ||||||||||  Journal:  Noninvasive and invasive mechanical ventilation for neurologic disorders. (Pubmed Central) -  Aug 30, 2022   
    This chapter outlines important general principles to consider when initiating, titrating, and discontinuing mechanical ventilation in patients with acute neurologic injuries. Important disease-specific considerations are also reviewed where appropriate.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Pricing, Journal:  A Critical Examination for the Pricing of Eculizumab and Efgartigimod in Myasthenia Gravis. (Pubmed Central) -  Aug 30, 2022   
    A second aspect of this commentary is to propose standards that should be set for the creation and evaluation of value claims in health technology assessment, in particular need fulfillment quality of life (QoL), that meet the demarcation test to distinguish science from non-science. The result is that the present ICER pricing claims for eculizumab and efgartigimod in myasthenia gravis should not be applied without consideration of more relevant evidence.
  • ||||||||||  Journal:  Learning Curve of Robot-Assisted Thymectomy: Single Surgeon's 7-Year Experience. (Pubmed Central) -  Aug 30, 2022   
    However, significantly higher hospital readmission at 30 days post surgery was recorded for myasthenic patients operated on during the first phase of the LC (2 cases vs. 0, p = 0.02). According to our data, LC in RAT seems to be steep, and RAT confirms to be safe even before reaching CLC.
  • ||||||||||  Journal:  Progressive motor quadriparesis with wasting: a diagnostic conundrum. (Pubmed Central) -  Aug 30, 2022   
    Carefully assessed clinical and electrophysiological markers which finally clinched the diagnosis of the rare disorder Lambert-Eaton myasthenic syndrome are elaborated. In the present context, recognition of the oddities on clinical and laboratory evaluation is of paramount importance to rule in causes of pure motor quadriparesis.
  • ||||||||||  Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
    Retrospective data, Journal:  Long-Term Remission With Low-Dose Rituximab in Myasthenia Gravis: A Retrospective Study. (Pubmed Central) -  Aug 26, 2022   
    Low-dose RTX infusions were sufficient to achieve undetectable CD19/20 cell counts and sustained clinical remission. In low and middle-income countries, the impact of low-dose RTX therapy represents a paradigm shift in decision-making for long-term treatment.
  • ||||||||||  Journal:  Immunodeficiency in patients with thymoma-associated myasthenia gravis. (Pubmed Central) -  Aug 24, 2022   
    Compared to group without immunodeficiency, they showed no significant differences in the severity of MG, significantly lower IgG concentrations and higher mortality rate. Thymoma-associated MG with immunodeficiency is a distinct subset requiring special attention to prevent infection during the follow-up period.
  • ||||||||||  Journal:  Pregnancy Outcomes in Patients with Congenital Myasthenic Syndromes. (Pubmed Central) -  Aug 24, 2022   
    The majority of women with CMS can safely plan pregnancy, but close follow-up is required from their neurology and obstetric teams. Although we identified no safety concerns, continued medication use should be reviewed on a case-by-case basis.
  • ||||||||||  Review, Journal:  Myasthenia gravis in clinical practice. (Pubmed Central) -  Aug 22, 2022   
    Knowledge of the peculiar aspects of their clinical and electrophysiological presentations is important for the diagnosis. Likewise, specific treatment and response time to each drug are crucial for proper care.