Myasthenia Gravis
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  • ||||||||||  Review, Journal:  Promising therapies for the treatment of myasthenia gravis. (Pubmed Central) -  Apr 28, 2024   
    Novel MG therapeutics belonging to the classes of complement inhibitors, neonatal Fc receptor (FcRn) inhibitors and B cell depletors, as well as the other emerging MG drugs in the pipeline constitute promising treatment strategies with potentially better efficacy and safety compared to the conventional MG treatments. However, further long-term research is needed in order to optimize the implementation of these new treatment options for the appropriate patient populations.
  • ||||||||||  Review, Journal, Checkpoint inhibition:  Immune Checkpoint Inhibitor-induced Myositis. (Pubmed Central) -  Apr 27, 2024   
    Diagnosis can generally be made on the basis of clinical presentation, elevation of muscle enzymes, and electromyographic changes, but some patients may require a muscle biopsy. The first line of therapy is high-dose corticosteroids, followed by immunosuppression, plasmapheresis, or intravenous immunoglobulin in patients with severe disease.
  • ||||||||||  Kesimpta (ofatumumab subcutaneous) / Novartis, Genmab, Ocrevus (ocrelizumab) / Roche, Biogen, Rituxan (rituximab) / Roche
    Journal, Adverse events:  Dental Adverse Effects of Anti-CD20 Therapies. (Pubmed Central) -  Apr 26, 2024   
    To our knowledge, this is the first case series reporting dental conditions developed in patients long-term treated with anti-CD20 treatments. This issue, literature data, and Vigilyze
  • ||||||||||  Review, Journal, Immunomodulating:  Novel immunomodulatory therapies in myasthenia gravis (Pubmed Central) -  Apr 26, 2024   
    This review explores new immunosuppressive therapies and ongoing clinical trials in MG, including depletion of B lymphocytes with agents such as rituximab and inebilizumab, as well as the use of eculizumab, efgartigimod, satralizumab, tocilizumab, and CAR-T (Chimeric Antigen Receptor-T) cell therapy. These advancements aim to improve disease control and patients' quality of life.
  • ||||||||||  CABA-201 / Cabaletta Bio
    Trial completion date, Trial initiation date, Trial primary completion date, CAR T-Cell Therapy:  CAB-201-004: RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Apr 25, 2024   
    P1/2,  N=12, Not yet recruiting, 
    The discoveries offer significant understanding into the molecular processes of TAMG and present possible indicators for categorizing risk. Trial completion date: Jul 2028 --> Sep 2029 | Initiation date: May 2024 --> Aug 2024 | Trial primary completion date: Jul 2028 --> Sep 2029
  • ||||||||||  Observational data, Journal, Adherence:  The analysis of factors affecting medication adherence in patients with myasthenia gravis: a cross-sectional study. (Pubmed Central) -  Apr 24, 2024   
    Meanwhile, the necessity of talking medicine and self-efficacy for taking medications under difficult circumstances are the protective factors for medication adherence. Our findings can help medical staff to enhance patients' medication adherence by informing patients necessary medical knowledge, emphasizing the necessity for medication, relieving patients' concerns regarding medication, and improving the self-efficacy for taking medications under difficult circumstances.
  • ||||||||||  Trial primary completion date:  The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis (clinicaltrials.gov) -  Apr 24, 2024   
    P=N/A,  N=144, Recruiting, 
    Our findings can help medical staff to enhance patients' medication adherence by informing patients necessary medical knowledge, emphasizing the necessity for medication, relieving patients' concerns regarding medication, and improving the self-efficacy for taking medications under difficult circumstances. Trial primary completion date: Mar 2024 --> Mar 2025
  • ||||||||||  Preclinical, Journal:  Change of voltage-gated sodium channel repertoire in skeletal muscle of a MuSK myasthenia gravis mouse model. (Pubmed Central) -  Apr 23, 2024   
    This was not explained by upregulation of Scn5a (the NaV1.5 gene), lowered muscle fibre firing thresholds or histologically detectable upregulated NaV1.5 channels. It remains to be established which factors are responsible for the observed ?-Conotoxin-GIIIB insensitivity and whether the NaV repertoire change is compensatory beneficial or a bystander effect.
  • ||||||||||  Journal:  Kearns-Sayre Syndrome Masquerading as Myasthenia Gravis. (Pubmed Central) -  Apr 23, 2024   
    We report a case of KSS found to have a novel large-scale mitochondrial DNA deletion. The presence of a mitochondrial pigmentary retinopathy found on dilated examination led to reconsideration of the previous diagnosis of myasthenia gravis and ultimately led to the correct diagnosis of KSS.
  • ||||||||||  Review, Journal:  Autoimmunity in thymic epithelial tumors: a not yet clarified pathologic paradigm associated with several unmet clinical needs. (Pubmed Central) -  Apr 21, 2024   
    The aim of this review is to provide the state of art in regard to the histological features of the different TET histotype, to the role of the different immune cells infiltrating tumor microenvironments and their impact in the break of central immunologic thymic tolerance in thymomas. We discuss here both cellular and molecular immunologic mechanisms inducing the onset of autoimmunity in TETs, limiting the portfolio of therapeutic strategies against TETs and greatly impacting the prognosis of associated autoimmune diseases.
  • ||||||||||  Review, Checkpoint inhibition:  Prognosis of immune checkpoint inhibitor-induced myasthenia gravis: a single center experience and systematic review. (Pubmed Central) -  Apr 18, 2024   
    Overlap of myositis with MG (OR?=?3.148, p?=?0.009) and anti-AChR antibody positivity (OR?=?3.364, p?=?0.005) were both significantly associated with poor outcomes. Our study reveals the prognosis of ICI-induced MG and suggests that myositis and/or myocarditis are severe comorbidities of ICI-induced MG, emphasizing the importance of early diagnosis and clinical intervention.
  • ||||||||||  Retrospective data, Journal, Adverse events, Checkpoint inhibition:  Immune checkpoint inhibitor related myositis: an observational, retrospective, pharmacovigilance study. (Pubmed Central) -  Apr 17, 2024   
    The proportion of fatal cases was significantly higher for myositis accompanied by myocarditis, severe myasthenia gravis, and malignant neoplasm progression compared to non-fatal cases.[Figure: see text]. Clinicians should be aware of the potential for ICI-associated myositis, as it can be particularly life-threatening, especially in elderly patients or when it occurs concurrently with other AEs such as myocarditis or severe myasthenia gravis.
  • ||||||||||  Journal:  Monoclonal antibodies in neuro-ophthalmology. (Pubmed Central) -  Apr 17, 2024   
    Further research into neuro-ophthalmologic disease mechanisms will provide accurate and specific targeting of important disease mediators through applications of future mAbs. As our understanding of these diseases and the relevant therapeutic targets evolve, we will continue to build on our understanding of how mAbs interfere with disease pathogenesis, and how these changes improve clinical outcomes and quality of life for patients.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    Review, Journal:  Eculizumab in myasthenia gravis: A review. (Pubmed Central) -  Apr 17, 2024   
    Current data support eculizumab as an effective, safe, and tolerable drug in cases of refractory MG. However, its cost can prevent it from being widely accessible to a majority of the general population.
  • ||||||||||  Preclinical, Journal, CAR T-Cell Therapy:  B cell lineage reconstitution underlies CAR-T cell therapeutic efficacy in patients with refractory myasthenia gravis. (Pubmed Central) -  Apr 17, 2024   
    By tracking the temporal evolution of CAR-T phenotypes, we demonstrated that proliferating cytotoxic-like CD8 clones were the main effectors in autoimmunity, whereas compromised cytotoxic and proliferation signature and profound mitochondrial dysfunction in CD8+ Te cells before infusion and subsequently defect CAR-T cells after manufacture might explain their characteristics in these patients. Our findings may guide future studies to improve CAR T-cell immunotherapy in autoimmune diseases.
  • ||||||||||  Review, Journal:  Inappropriate placement of urinary catheters into the ureter: A case report and literature review. (Pubmed Central) -  Apr 16, 2024   
    Female gender, neurologic conditions, and long-term catheterization were identified as predominant risk factors. Early detection of ureteral cannulation can prevent severe complications, particularly in certain special populations such as patients with neurogenic bladder or SCI, who may have reduced sensation and expression capabilities.
  • ||||||||||  Review, Journal:  The intricate dance of non-coding RNAs in myasthenia gravis pathogenesis and treatment. (Pubmed Central) -  Apr 16, 2024   
    This comprehensive review consolidates the landscape of dysregulated ncRNAs in MG, briefly delineating their pivotal role in MG pathogenesis. Furthermore, it explores their promise as prospective biomarkers, aiding in the elucidation of disease subtypes, assessment of disease severity, monitoring therapeutic responses, and as novel therapeutic targets.
  • ||||||||||  Journal:  Markedly upregulated serum semaphorin 4A as a novel activity marker of myasthenia gravis. (Pubmed Central) -  Apr 16, 2024   
    Serum Sema4A levels were correlated with AChR antibody levels (Spearman's ??=?0.39, p?=?0.03) and MG Foundation of America clinical classification classes (Spearman's ??=?0.38, p?=?0.04) in patients with AChR antibody-positive MG. In conclusion, high serum Sema4A levels may reflect T-cell activation, and this molecule could be a potential marker of disease activity in MG.
  • ||||||||||  FDA event, Review, Journal:  Structure and function of therapeutic antibodies approved by the US FDA in 2023. (Pubmed Central) -  Apr 15, 2024   
    Of the approved antibodies, two targeted programmed cell death receptor-1 (PD-1) for orphan indications, two targeted CD20 for diffuse large B cell lymphoma, two targeted different receptors (B-cell maturation antigen [BCMA] and G-coupled protein receptor class C, group 5, member D [GPRC5D]) for treatment of multiple myeloma, and one each that targeted amyloid-? protofibrils for Alzheimer's disease, neonatal Fc receptor alpha-chain for myasthenia gravis, complement factor C5 for CD55 deficiency with hyper-activation of complement, angiopathic thrombosis and severe protein-losing enteropathy disease, interleukin (IL)-23p19 for severely active ulcerative colitis, IL-17A-F for plaque psoriasis and respiratory syncytial virus (RSV)-F protein for season-long RSV prophylaxis in infants.
  • ||||||||||  Review, Journal:  Myasthenia gravis-Gender aspects and family planning (Pubmed Central) -  Apr 15, 2024   
    Gender-specific factors can influence the course of MG. Adequate medical counselling and multidisciplinary collaboration are essential for MG patients who wish to have children.
  • ||||||||||  Journal:  Novel single-nucleus transcriptomics unravels developmental and functionally controlled principles of mammalian neuromuscular junctions. (Pubmed Central) -  Apr 15, 2024   
    Breaking the barriers between distinct research subdisciplines, this study opens new neurochemistry research directions that recombine developmental, regulatory, and functional transcriptomics in NMJ-including tissues. Moreover, these findings may facilitate tests of diverse pharmaceutical and therapeutic modulators of neuromuscular functioning in health and disease, assisting the translational research progress in treating devastating neuromuscular states such as in amyotrophic lateral sclerosis, myasthenia gravis or individuals poisoned occupationally or otherwise with anticholinesterase inhibitors.