Caregivers' Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy: A Comparative Study - PubMed
Source : https://pubmed.ncbi.nlm.nih.gov/37445216/
Background: The primary aim of this study was to explore current caregivers' expectations on possible functional changes following treatment in comparison to data obtained in the pre-pharmacological era. Methods: A...
Conclusions: The results indicate an overall more positive attitude both in terms of recent disease courses and expectations. Carers now perceive a higher chance of at least remaining stable as opposed to 2016 and although the great majority, regardless of age or functional level, would still consider participating in a clinical trial even if...
Roles of FcRn in Antigen-Presenting Cells during Autoimmunity and a Clinical Evaluation of Efgartigimod as an FcRn Blocker - PubMed
Source : https://pubmed.ncbi.nlm.nih.gov/37375507/
The immune system is a complex network of multiple cells, tissues, and organs that protects the body against foreign pathogenic invaders. However, the immune system may mistakenly attack healthy cells...
Relevance: This article aims to provide an overview of the importance of FcRn in antigen-presenting cells and its potential as a therapeutic target in autoimmune diseases, using efgartigimod as an example.
Neonatal Fc Receptor Inhibitor Therapeutics in... : Journal of Clinical Neuromuscular Disease
h autoantibody titers are reduced resulting in clinical improvement and disease abatement. The FcRn targeting mechanism is similar to that of intravenous immunoglobulin (IVIg) in which saturated FcRn facilitates accelerated...
Relevance: 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.
Myasthenia Gravis and Ischemic Stroke: A Bidirectional Mendelian Randomization Study - PubMed
Source : https://pubmed.ncbi.nlm.nih.gov/37403387/
Bidirectional MR analysis did not provide evidence to support a causal relationship between genetically predicted MG and IS, although observational studies have found such a potential link.
Conclusion: Bidirectional MR analysis did not provide evidence to support a causal relationship between genetically predicted MG and IS, although observational studies have found such a potential link.
Myasthenia gravis and independent risk factors for recurrent infection: a retrospective cohort study - BMC Neurology
Source : https://bmcneurol.biomedcentral.com/articles/10.1186/s12883-023-03306-3
Background Approximately 10% to 20% of myasthenia gravis (MG) patients have experienced a myasthenic crisis (MC), which contributes to morbidity and mortality. MC triggered by infection is associated with poor...
Conclusions: The independent risk factors for recurrent infections in MG patients identified in this study include the presence of concomitant diabetes mellitus, hypomagnesaemia, activated partial thromboplastin time prolongation, and longer duration of hospitalization, highlighting the need for targeted interventions to prevent recurrent...