Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT) - PubMed
Source : https://pubmed.ncbi.nlm.nih.gov/36598575/
There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as...
Conclusions/Relevance: The substantial and durable improvements in HRQoL end points in this study demonstrate the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.
A Digital Telehealth System to Compute Myasthenia Gravis Core Examination Metrics: Exploratory Cohort Study
Source : https://neuro.jmir.org/2023/1/e43387
Background: Telemedicine practice for neurological diseases has grown significantly during the COVID-19 pandemic. Telemedicine offers an opportunity to assess digitalization of examinations and enhances access to modern computer vision and...
Conclusions: We demonstrated the ability to objectively quantitate the MG-CE. Our results indicate that the MG-CE should be revisited to consider some of the new metrics that our algorithm identified. We provide a proof of concept involving the MG-CE, but the method and tools developed can be applied to many neurological disorders and have...
In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy - PubMed
Source : https://pubmed.ncbi.nlm.nih.gov/37451880/
Globally, adeno-associated virus (AAV) vectors have been increasingly used for clinical gene therapy trials. In Australia, AAV-based gene therapy is available for hereditary diseases such as retinal dystrophy or spinal...
Relevance: Globally, adeno-associated virus (AAV) vectors have been increasingly used for clinical gene therapy trials. In Australia, AAV-based gene therapy is available for hereditary diseases such as retinal dystrophy or spinal muscular atrophy 1 (SMA1).
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.