Myasthenia Gravis
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  • ||||||||||  Journal:  Clinical characteristics of late-onset myasthenia gravis. (Pubmed Central) -  Apr 10, 2024   
    The incidence of LOMG with thymoma was low. Supramaximal repetitive nerve stimulation (Jolly test) of the facial nerve was the easiest to detect and Jolly test of Class IIb was tested in the highest positive rate and Class I was in the lowest one.
  • ||||||||||  Zilbrysq (zilucoplan) / UCB
    Journal:  Zilucoplan (Zilbrysq) for myasthenia gravis. (Pubmed Central) -  Apr 9, 2024   
    Supramaximal repetitive nerve stimulation (Jolly test) of the facial nerve was the easiest to detect and Jolly test of Class IIb was tested in the highest positive rate and Class I was in the lowest one. No abstract available
  • ||||||||||  Observational data, Journal:  The epidemiology and societal costs of myasthenia gravis in (Pubmed Central) -  Apr 9, 2024   
    The incidence and prevalence of MG are higher than previously estimated, and the total societal costs of MG are substantial. Our findings demonstrate that productivity losses, and the value of LLY and HRQoL constitute a considerable proportion of the total societal costs.
  • ||||||||||  Journal:  Empirical Pyridostigmine in a Patient with Difficult Weaning from Mechanical Ventilation after Traumatic Brain Injury. (Pubmed Central) -  Apr 8, 2024   
    Novel treatments are promising, though subject to restricted access due to costs. "Hidden" cranial injuries may account for subtle bulbar symptoms in victims of traumatic brain injury and should be searched for.Myasthenia gravis has been reported in association with trauma, which comes first and is often difficult to ascertain.A trial of pyridostigmine may be reasonable in difficult-to-wean patients when all other causes have been excluded for the benefit of the doubt.
  • ||||||||||  bleomycin / Generic mfg.
    Journal:  Contribution of collagen XIII to lung function and development of pulmonary fibrosis. (Pubmed Central) -  Apr 7, 2024   
    We suggest that respiratory muscle myasthenia is the primary cause of the breathing problems suffered by CMS19 patients in addition to skeletal deformities. Induction of collagen XIII expression in the IPF patients' lungs warrants further studies to reveal collagen XIII-dependent disease mechanisms.
  • ||||||||||  Triesence (triamcinolone acetonide injectable suspension) / Novartis
    Journal:  Peribulbar Corticosteroids for Ocular Myasthenia Gravis. (Pubmed Central) -  Apr 5, 2024   
    Peribulbar corticosteroids can be effective in ocular myasthenia gravis. We suggest that longer-acting agents such as triamcinolone are preferable, to reduce injection frequency.
  • ||||||||||  Review, Journal:  Single breath count test and its applications in clinical practice: a systematic review. (Pubmed Central) -  Apr 5, 2024   
    Also, Single breath count correlated significantly with forced expiratory volume in 1 sec (FEV1) and forced vital capacity (FVC) in children with pulmonary pathology and in patients with COVID-19 it was used to rule out the need for noninvasive respiratory support. SBCT will undoubtedly be an asset in low resource settings and in tele-medicine to assess the prognosis and guide management of different respiratory and neuromuscular diseases.
  • ||||||||||  Journal:  Myasthenia Gravis in Pregnancy and the Newborn. (Pubmed Central) -  Apr 4, 2024   
    SBCT will undoubtedly be an asset in low resource settings and in tele-medicine to assess the prognosis and guide management of different respiratory and neuromuscular diseases. No abstract available
  • ||||||||||  Journal:  The severity of MUSK pathogenic variants is predicted by the protein domain they disrupt. (Pubmed Central) -  Apr 3, 2024   
    We analyze known pathogenic variants in MUSK to discover domain-specific genotype-phenotype correlations; variants that lead to a loss of protein expression, disruption of the Ig1 domain, or Dok-7 binding are associated with the most severe phenotypes. A conceptual model is provided to explain the severe phenotypes seen in Ig1 variants and the poor response of our patient to pyridostigmine.
  • ||||||||||  Journal:  Myasthenia Gravis in Pregnancy and the Newborn. (Pubmed Central) -  Mar 31, 2024   
    A larger population, rigorous study design and conduction, standardized interventions and outcomes, and standardized reporting are essential. No abstract available
  • ||||||||||  Review, Journal:  MuSK Myasthenia Gravis-Potential Pathomechanisms and Treatment Directed against Specific Targets. (Pubmed Central) -  Mar 31, 2024   
    It is characterized by a more severe clinical course, more frequent complications, and often inadequate response to treatment. Here, we review the current state of knowledge about potential pathomechanisms of the MuSK-MG and their therapeutic implications as well as ongoing research in this field, with reference to key points of immune-mediated processes involved in the background of myasthenia gravis.
  • ||||||||||  KYV-101 / Kyverna
    Journal, CAR T-Cell Therapy:  CD19-targeted chimeric antigen receptor T cell therapy in two patients with multiple sclerosis. (Pubmed Central) -  Mar 30, 2024   
    CAR-T cell presence and expansion were observed in the cerebrospinal fluid without clinical signs of neurotoxicity, which, along with intrathecal antibody reduction, indicates expansion-dependent effects of CAR-T cells on CD19+ target cells in the CNS. Larger clinical studies assessing CD19 CAR-T cells in MS are warranted.
  • ||||||||||  Defining Immunogenomic Signatures in B Cell-Mediated Autoimmunities (Exhibit Hall F1; Poster Board Number: B605) -  Mar 29, 2024 - Abstract #IMMUNOLOGY2024IMMUNOLOGY_2227;    
    Additionally, statistically significant differences (p-value=0.05) in IGHV gene usage within IgG subisotypes were evident across each disease-specific group, despite small initial sample numbers. Collectively, with these data we are beginning to define how IGH genomic variation may be associated with B cell autoimmunities.
  • ||||||||||  LONG-ACTING DERIVATIVES OF A BACTERIAL IGG PROTEASE WITH REDUCED IMMUNOGENICITY FOR THE RAPID DEGRADATION OF IGG AUTOANTIBODIES (Poster View) -  Mar 29, 2024 - Abstract #EULAR2024EULAR_2655;    
    P2
    Given its ability to address multiple pathogenic mechanisms as a single drug, it has the potential to achieve improved clinical outcomes in autoantibody-mediated diseases. The superior pharmacokinetic and pharmacodynamic properties of the engineered IdeS variants support their potential for the rapid and sustained degradation of IgG autoantibodies, while their reduced immunogenicity based on ex vivo assessments suggests redosing will be possible for long-term management of recurrent flares or chronic autoimmune diseases.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio, Rituxan (rituximab) / Roche
    NOVEL DRUG USE IN P-ANCA VASCULITIS WITH SEVERE CUTANEOUS DISEASE (Strauss 2) -  Mar 29, 2024 - Abstract #EULAR2024EULAR_1333;    
    To our knowledge this is the first reported case of AAV treated with efgartigimod. Further research is needed to define its role in managing AAV.
  • ||||||||||  KYV-201 / Kyverna, Intellia Therap
    PRECLINICAL DEVELOPMENT OF KYV-201, AN INVESTIGATIONAL ALLOGENEIC ANTI-CD19 CAR T CELL FOR THE TREATMENT OF AUTOIMMUNE DISEASE (Poster View) -  Mar 29, 2024 - Abstract #EULAR2024EULAR_744;    
    KYV-201 combines the same fully human anti-CD19 CAR construct as KYV-101, an autologous CAR T-cell therapy being studied in lupus nephritis, systemic sclerosis, myasthenia gravis, and multiple sclerosis, with a differentiated allogeneic platform developed by Intellia Therapeutics involving selected CRISPR/Cas9-mediated gene edits[2,5]. The preclinical data generated for KYV-201 demonstrates the functionality and safety necessary to advance to first-in-human studies, bringing the promise of allogeneic CD19 CAR T cells for autoimmune diseases closer to fruition.
  • ||||||||||  Review, Journal, Surgery:  Thymus Surgery Prospectives and Perspectives in Myasthenia Gravis. (Pubmed Central) -  Mar 28, 2024   
    Videothoracoscopic surgery or robotic surgery led to a decrease in the length of hospital stay for these patients. This paper aims to synthesize the information presented in the literature in order to create a background for the perspectives of thymectomy.
  • ||||||||||  Review, Journal:  Therapeutic potential of natural killer cells in neuroimmunological diseases. (Pubmed Central) -  Mar 27, 2024   
    In this review, we summarize the evidence for the heterogenic involvement of NK cells in the above conditions. Further, we describe cutting-edge NK-cell-based immunotherapy for neuroimmunological diseases in preclinical and clinical development and highlight challenges that must be overcome to fully realize the therapeutic potential of NK cells.
  • ||||||||||  Enrollment open, Trial completion date, Trial primary completion date:  Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Myasthenia Gravis (clinicaltrials.gov) -  Mar 26, 2024   
    P=N/A,  N=16, Recruiting, 
    Further, we describe cutting-edge NK-cell-based immunotherapy for neuroimmunological diseases in preclinical and clinical development and highlight challenges that must be overcome to fully realize the therapeutic potential of NK cells. Active, not recruiting --> Recruiting | Trial completion date: Jul 2024 --> Dec 2024 | Trial primary completion date: May 2024 --> Nov 2024