Myasthenia Gravis
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  • ||||||||||  Veopoz (pozelimab-bbfg) / Regeneron
    Review, Journal:  Pozelimab: First Approval. (Pubmed Central) -  Nov 6, 2023   
    Pozelimab is also undergoing clinical development in several other countries worldwide for the treatment of CD55-deficient PLE, PNH and myasthenia gravis. This article summarizes the milestones in the development of pozelimab leading to this first approval for the treatment of adults, and paediatric patients aged ??1 year with CD55-deficient PLE, also known as CHAPLE disease, in the USA.
  • ||||||||||  Retrospective data, Journal:  Myasthenic crisis in thymoma-associated myasthenia gravis: a multicenter retrospective cohort study. (Pubmed Central) -  Nov 6, 2023   
    This article summarizes the milestones in the development of pozelimab leading to this first approval for the treatment of adults, and paediatric patients aged ??1 year with CD55-deficient PLE, also known as CHAPLE disease, in the USA. Within the TMG group, the hospital stay was significantly longer in patients with unresected thymoma compared to that in postoperative myasthenic crisis (POMC) (47.68
  • ||||||||||  Journal:  Disease-Based Prognostication: Myasthenia Gravis. (Pubmed Central) -  Nov 6, 2023   
    Our objective is to provide a framework that can assist a clinician who encounters the MG patient for the first time and attempts to prognosticate probable outcomes in individual patients. In this review article, we explore clinical type, age of onset, antibody status, severity of disease, thymus pathology, autoimmune, and other comorbidities as prognostic factors in MG.
  • ||||||||||  Preclinical, Journal:  Sodium butyrate alleviates R97-116 peptide-induced myasthenia gravis in mice by improving the gut microbiota and modulating immune response. (Pubmed Central) -  Nov 5, 2023   
    GO enrichment and KEGG pathway analysis unveiled that the function of these DEGs was mainly focused on immunoglobulin production, mitochondrial respiratory chain complex, ribosome, oxidative phosphorylation, and CNS diseases including amyotrophic lateral sclerosis. We have found that butyrate was significantly reduced in MG patients and NaB gavage could evidently improve MG symptoms in EAMG mice by changing the gut microbiota, regulating the immune response, and altering the gene expression and function of B cells, suggesting NaB might be a potential immunomodulatory supplement for MG drugs.
  • ||||||||||  Journal:  A 50 year old man with progressive weakness in lower limbs (Pubmed Central) -  Nov 4, 2023   
    Treatment with symtomatic and/or immunosuppressive therapy is important as well as a workup for possible malignancy. This article identifies the clinical features, diagnosis and treatment of this uncommon disease.
  • ||||||||||  Scalable human NMJ-on-a-chip (WCC Halls A-C) -  Nov 3, 2023 - Abstract #Neuroscience2023NEUROSCIENCE_6456;    
    Owing to the unique open-top device design, we also scaled this device to a standardized 96-well microplate format to suit high-throughput screening modalities based on high-content imaging and automated liquid handling systems. Together, the NMJ-on-a-Chip
  • ||||||||||  Proteolytic ectodomain shedding of muscle-specific tyrosine kinase in myasthenia gravis (WCC Halls A-C) -  Nov 3, 2023 - Abstract #Neuroscience2023NEUROSCIENCE_4703;    
    The results may explain why MuSK-MG is refractory and causes rapid muscle atrophy in some patients due to the denervation associated with Ab-induced disruption of neuromuscular transmission at the NMJ. Such discoveries pave the way for new MG treatments, and MuSK may be used as a biomarker for other neuromuscular diseases in preclinical studies, clinical diagnostics, therapeutics, and drug discovery.
  • ||||||||||  Journal:  Ocular myasthenia gravis (Pubmed Central) -  Nov 1, 2023   
    The article discusses modern approaches to the treatment of the ocular myasthenia gravis. The authors present clinical observations of 2 patients with ocular myasthenia gravis.
  • ||||||||||  Biomarker, Review, Journal:  Current Biomarker Strategies in Autoimmune Neuromuscular Diseases. (Pubmed Central) -  Oct 31, 2023   
    Collectively, biomarkers have the potential to improve the management of inflammatory neuromuscular disorders. However, novel strategies and further research are needed to establish clinically meaningful biomarkers.
  • ||||||||||  Enrollment closed, Trial primary completion date:  A Prospective, Single-Center Investigation of the da Vinci SP (clinicaltrials.gov) -  Oct 31, 2023   
    P=N/A,  N=15, Active, not recruiting, 
    However, novel strategies and further research are needed to establish clinically meaningful biomarkers. Recruiting --> Active, not recruiting | Trial primary completion date: Jul 2023 --> Apr 2024
  • ||||||||||  Journal:  Downregulation of HMGB1 in thymoma cells affects T cell differentiation. (Pubmed Central) -  Oct 30, 2023   
    Co-culture of Thy0517 and PBMC showed that the percentage of Th17 cells in PBMC was lower than that in the control group, and the percentage of Treg cells was higher than that in the control group. These findings demonstrate that HMGB1 is involved in the mechanism of abnormal Th17/Treg cell differentiation in thymoma and affects the occurrence of immune abnormalities in MG-associated thymoma.
  • ||||||||||  Review, Journal:  Eyes and the heart: what a clinician should know. (Pubmed Central) -  Oct 30, 2023   
    Rarer diseases, such as Fabry disease, would be accompanied by ocular signs such as cornea verticillata and such cardiac manifestations include cardiac hypertrophy as well as arrhythmias. This review examines the interplay between the eye and the cardiovascular system and emphasises the use of conventional and emerging tools to improve diagnosis, management and prognostication of patients.
  • ||||||||||  Review, Journal:  Usefulness and Clinical Impact of Whole-Body MRI in Detecting Autoimmune Neuromuscular Disorders. (Pubmed Central) -  Oct 28, 2023   
    It can also be used as a biomarker, detect the pattern of specific muscle involvement, and is a useful tool for targeting the optimal muscle site for biopsy. In this work, we summarized the most used technical protocol of whole-body MRI and the role of this imaging technique in autoimmune neuromuscular disorders.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD), Opdivo (nivolumab) / Ono Pharma, BMS, Yervoy (ipilimumab) / Ono Pharma, BMS
    Journal, Adverse events, Checkpoint inhibition:  Musculoskeletal adverse events induced by immune checkpoint inhibitors: a large-scale pharmacovigilance study. (Pubmed Central) -  Oct 26, 2023   
    Furthermore, the combination therapy of nivolumab and ipilimumab does not result in a statistically significant escalation of the risk associated with the major musculoskeletal adverse events. Immune checkpoint inhibitors administration triggers a range of musculoskeletal adverse events, warranting the optimization of their management during clinical practice.
  • ||||||||||  Journal:  Maternal immunoglobulin treatment can reduce severity of fetal acetylcholine receptor antibody-associated disorders (FARAD). (Pubmed Central) -  Oct 26, 2023   
    Immune checkpoint inhibitors administration triggers a range of musculoskeletal adverse events, warranting the optimization of their management during clinical practice. These observations highlight that FARAD is an important differential diagnosis of genetically determined congenital neuromuscular disorders even in asymptomatic mothers, and that IVIG therapy during the pregnancy has the potential to improve the outcome of the children.
  • ||||||||||  Enrollment change, Trial primary completion date:  Baby Detect : Genomic Newborn Screening (clinicaltrials.gov) -  Oct 25, 2023   
    P=N/A,  N=6000, Recruiting, 
    Most patients are expected to experience partial/complete improvement within 1 month. N=40000 --> 6000 | Trial primary completion date: Aug 2023 --> Aug 2025
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Journal:  Efgartigimod improves triple-negative myasthenia gravis. (Pubmed Central) -  Oct 25, 2023   
    The introduction of complement inhibition will considerably change the traditional therapeutic strategy for MG. No abstract available
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca, Zilbrysq (zilucoplan) / UCB
    Review, Journal:  Role of complement in myasthenia gravis. (Pubmed Central) -  Oct 24, 2023   
    Complement therapies were initially studied and approved for paroxysmal nocturnal hemoglobinuria and in the past decade, those have also been studied in myasthenia gravis. The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD), Tecentriq (atezolizumab) / Roche
    Journal, Adverse events, Checkpoint inhibition:  Severe neuromuscular immune-related adverse events of immune checkpoint inhibitors at national cancer center in Korea. (Pubmed Central) -  Oct 23, 2023   
    The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review. In this real-world monocentric study, ICI-induced neuromuscular irAEs were rare but potentially devastating; thus, physicians should remain vigilant to enable prompt recognition and management of irAEs.
  • ||||||||||  Journal, Video, Surgery:  Validity of Video-assisted Thoracic Surgery for Thymoma (Pubmed Central) -  Oct 22, 2023   
    Liga Sure Impact was useful to remove a large thoracic wall tumor with VATS for resection of thick muscle. VATS thymectomy and VATS extended thymectomy for patients with thymoma may be effective methods to improve not only prognosis but also MG, provided the range of resections are comparable to that of conventional open surgery.
  • ||||||||||  Journal:  Multiple sclerosis and autoimmune diseases - a case control study. (Pubmed Central) -  Oct 22, 2023   
    Here, we summarize the potential role of circulating miRNAs in different subgroups of MG to promote personalized medicine. Our study revealed that the probability of autoimmune diseases co-occurring in patients with MS, and in their relatives, is higher and we found the greatest risk to be for Hashimoto's thyroiditis.
  • ||||||||||  Nucynta (tapentadol) / Collegium Pharma, Grunenthal
    Clinical, Journal:  Dual opioid therapy in a cancer patient with myasthenia gravis who developed respiratory depression from codeine. (Pubmed Central) -  Oct 22, 2023   
    This review outlines the currently reported histogenetic phenotypes of TETs, including those related to thymoma-associated myasthenia gravis, summarizes their genetic signatures, and provides a perspective for the future direction of TET classification. This case report describes a patient who was referred to our unit after an emergency room admission for respiratory depression induced by codeine, and was successfully managed with tapentadol.
  • ||||||||||  Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
    Review, Journal:  Rituximab treatment in myasthenia gravis. (Pubmed Central) -  Oct 18, 2023   
    Moreover, few evidence has been reported in seronegative MG and juvenile MG and some questions about regimen of administration or monitoring strategies, remains open. In this review, we intend to revise the available literature on this topic and resume the current evidence of effectiveness of Rituximab in MG, with special attention to results on every MG subtype, as well as the administration protocols, monitoring strategies and safety profile of the drug.
  • ||||||||||  prednisone / Generic mfg.
    Journal, Metabolomic study:  Serum metabolomics of treatment response in myasthenia gravis. (Pubmed Central) -  Oct 14, 2023   
    Metabolomic profiles of outcome measures had limited overlap consistent with their assessing distinct aspects of treatment response and supporting unique biological underpinning for each outcome measure. Interindividual variation in prednisone metabolism may be a determinate of how well patients respond to treatment.
  • ||||||||||  Enrollment closed, Trial primary completion date:  Acupuncture in Myasthenia Gravis (AcuMG) (clinicaltrials.gov) -  Oct 13, 2023   
    P=N/A,  N=20, Active, not recruiting, 
    Interindividual variation in prednisone metabolism may be a determinate of how well patients respond to treatment. Recruiting --> Active, not recruiting | Trial primary completion date: Sep 2023 --> Dec 2023
  • ||||||||||  Rituxan (rituximab) / Roche
    Journal:  Rituximab as a sole steroid-sparing agent in generalized myasthenia gravis: Long-term outcomes. (Pubmed Central) -  Oct 13, 2023   
    Recruiting --> Active, not recruiting | Trial primary completion date: Sep 2023 --> Dec 2023 The present study supports that rituximab is an effective and well tolerated treatment for refractory anti-AChR?+?and DSN MG patients, while anti-MuSK?+?remains the group experiencing the greater benefits.
  • ||||||||||  Tai'ai (telitacicept) / Rongchang Pharma
    Enrollment open:  Study of Telitacicept in Generalized Myasthenia Gravis (clinicaltrials.gov) -  Oct 12, 2023   
    P3,  N=100, Recruiting, 
    Our proposed ML model can identify several taxonomic markers and effectively discriminate patients with MG from those without with a high accuracy, the ML strategy can be applied as a benchmark to conduct noninvasive screening of MG. Not yet recruiting --> Recruiting