Myasthenia Gravis
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  • ||||||||||  Journal, IO biomarker:  Expression of lymphocyte activation gene-3 on CD4+T cells is regulated by cytokine interleukin-18 in myasthenia gravis. (Pubmed Central) -  Mar 8, 2024   
    These results suggest that IL-18 may regulate the expression pattern of mLAG-3 in CD4+T cells and sLAG-3 via ADAM10- and ADAM17-mediated pathways, thus affecting the immune effects of CD4+T cells. This study provides a preliminary exploration of the upstream regulatory molecules of the LAG-3 and IL-18/LAG-3 signalling pathways for potential targeted therapy of autoimmune diseases in the future.
  • ||||||||||  PLASMAPHERESIS IN NEUROLOGICAL DISEASES PERFORMED BY NEPHROLOGISTS (SAT-304; Exhibition Hall and Main Foyer) -  Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1777;    
    All patients exhibited a favorable response, resulting in improved short-term survival. Nephrologists should promote the use of this technique and document the results to expand the level of evidence in clinical practice.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD)
    ATYPICAL HEMOLYTIC SYNDROME INDUCED BY PEMBROLIZUMAB. MANAGEMENT AND OUTCOMES (SUN-005; Exhibition Hall and Main Foyer) -  Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_688;    
    Contrary to the prior reports of Pembrolizumab related MAHA and HLH, use of corticosteroids and PLEX was not effective presumably due to the different mechanisms of immune injury. Prompt response to Eculizumab with complete renal and hematologic recovery points to the novel pathway of how Pembrolizumab can affect the immune system on the level of complement regulatory proteins.
  • ||||||||||  Evidence Gap Analysis of the Burden of Illness and Treatment of Myasthenia Gravis () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2036;    
    Besides traditional non-specific immunosuppressive agents, recently available biologics (e.g., eculizumab, ravulizumab, efgartigimod, rozanolixizumab, zilucoplan) are increasingly used in MG to target specific components of the immune pathway. We identified several gaps in the literature, including the need for comprehensive studies to advance our understanding of the epidemiology, humanistic and economic burden, as well as the evolving treatment pathways in MG.
  • ||||||||||  Rystiggo (rozanolixizumab-noli) / UCB, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Cost per Responder for FcRn Inhibitors for Generalized Myasthenia Gravis () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2026;    
    A systematic literature review identified relevant randomised controlled studies in gMG which allowed for a network meta-analysis (NMA) to be performed for rozanolixizumab 7mg/kg, rozanolixizumab 10mg/kg, and efgartigimod 10mg/kg. While the FcRn inhibitor class were shown to have similar responder rates, rozanolixizumab showed lower cost per responder based on gMG patients
  • ||||||||||  Advancing Patient-Centered Outcomes Research for Rare Diseases: A Mixed-Methods Approach () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_1595;    
    Understanding patient-centered outcomes across various rare diseases not only addresses uncertainties in the evidence base but also alleviates the burden on researchers tasked with defining outcome measures for each individual disease. Moreover, this approach has the potential to expedite cross-cutting research, offering deeper insights into patient preferences and enhancing the value assessment of emerging technologies for rare diseases.
  • ||||||||||  Comorbidities in Myasthenia Gravis in the US National Veterans Affairs Health Network. () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_1528;    
    Patients diagnosed with MG were at significant risk to develop comorbidities commonly associated with MG and the onset occurred at a faster rate compared to the non-MG cohort. These findings underscore the association between MG and an elevated risk of specific comorbidities.
  • ||||||||||  BHV-1300 / Biohaven
    Novel Bispecific Degrader BHV-1300 Achieves Rapid, Robust, and Selective IgG Reduction in Preclinical Models Including Nonhuman Primates (Colorado Convention Center | 108/110/112) -  Mar 8, 2024 - Abstract #AAN2024AAN_4656;    
    This compares favorably to published data for FcRn inhibitor IgG reducing therapies; BHV-1300 has important differentiating properties: speed of onset, shortened time to maximal effect, depth of IgG lowering, ability to be co-administered with biologics and target-related safety. Coupled with other potential mechanistic advantages (brief period of exposure; lack of effects on albumin, cholesterol, or triglycerides; potential for reduced immunosuppression; and compatibility with Fc-containing biologics), BHV-1300 represents the first member of a new drug class with potentially significantly improved benefit-risk for neuroinflammatory and autoimmune disorders including acute manifestations (MG crises/flares) or chronic disease (MG, rheumatoid arthritis, etc).
  • ||||||||||  Simultaneous Occurrence of Myositis and Myasthenia Gravis (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4596;    
    The key to accurate diagnosis of BCIM echoed throughout the literature remains muscle biopsy. Aggressive treatment using IVIg followed by high dose steroids (i.e., prednisone) led to dramatic improvement and a favorable neurologic outcome in our patient and in many other patient cases described in the literature.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    A Unique Case of Non-paraneoplastic Lambert-Eaton Myasthenic Syndrome Treated with Subcutaneous Immunoglobulin (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4595;    
    For LEMS patients refractory to initial symptomatic treatment with amifampridine, immunomodulatory therapy with intravenous immunoglobulin (IVIG) is often utilized...The diagnosis of non-paraneoplastic LEMS requires a high level of clinical suspicion, and prompt diagnosis is necessary to facilitate treatment. Subcutaneous immunoglobulin therapy may be a reasonable alternative for patients with LEMS who do not tolerate the intravenous formulation.
  • ||||||||||  Poteligeo (mogamulizumab-kpkc) / Kyowa Kirin
    Case Report: Mogamulizumab-associated Myositis (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4590;    
    Subcutaneous immunoglobulin therapy may be a reasonable alternative for patients with LEMS who do not tolerate the intravenous formulation. Neuromuscular irAEs may be seen in association with mogamulizumab therapy due to Treg depletion and the associated dysregulation of cytotoxic T-cell activity.
  • ||||||||||  Development of Myasthenia Gravis 6 Years After Thymectomy (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4588;    
    Currently, only CT thorax is recommended for surveillance after thymoma resection. The presence of a new onset of myasthenia gravis in the absence of indicators of thymoma recurrence within the thorax should prompt additional investigations for distant metastasis.
  • ||||||||||  Acetylcholine Binding Receptor Positivity from Passive Antibody Transfer in a Case of Progressive Muscular Atrophy (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4585;    
    However, the presence of ocular symptoms, coupled with extra-ocular movement impairment that responded to plasma-exchange, led us to suspect symptomatic myasthenia gravis caused by passive antibody transfer from IVIG. While passive antibody transfer post-IVIG has been reported for other antibodies, it has not been described for AchRB-ab.
  • ||||||||||  prednisone / Generic mfg.
    Coexistence of Acetylcholine Receptor (AChR) Antibody Positive Myasthenia Gravis in Two Patients with Hereditary Myopathies Associated with Extraocular Muscle Weakness (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4577;    
    Patient 2 is a 61-year-old man who presented with bilateral ptosis and diplopia since his teens followed later by dysarthria and dysphagia that only mildly improved with pyridostigmine and prednisone...Patients with hereditary myopathies associated with EOM weakness may be at increased risk for MG due to muscle fiber degeneration, loss of immune tolerance, and consequent generation of AChR antibodies5. Clinicians should consider appropriate genetic testing in patients with seropositive MG and EOM weakness that fails to improve with standard MG therapies.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Real-world Reduction in Oral Corticosteroid Utilization Following Efgartigimod Initiation in Patients Living with Generalized Myasthenia Gravis (Colorado Convention Center | Mile High 2/3) -  Mar 8, 2024 - Abstract #AAN2024AAN_4404;    
    However, patients with SCLC and ANNA1/Hu-IgG PNS survived longer than those without PNS, possibly due to earlier cancer detection or increased antitumor immunity. While additional data is required to assess longer-term patterns, early insights into OCS utilization post-efgartigimod initiation in patients with gMG indicate that a substantial proportion experienced reduction in OCS usage over the first 6 months post-efgartigimod initiation.
  • ||||||||||  Investigating Immunological Profiles of Thymus in Myasthenia Gravis by scRNA-seq (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4260;    
    Our study highlights the presence of an immune cell imbalance between the thymus and peripheral circulation in MG patients. Further investigations are necessary to confirm the underlying mechanisms and elucidate the role of these imbalanced immune cells in the pathogenesis of MG.
  • ||||||||||  Rituxan (rituximab) / Roche
    Management of Myasthenia Gravis Around the Globe: Consensus Guidelines vs. Realities of Practice (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4258;    
    Despite guidelines, only 54% of respondents recommended thymectomy in someone with AChR-MG, only 25% recommended vaginal delivery in someone with well controlled MG, and only 27% recommended early initiation of rituximab in someone with muscle-specific tyrosine kinase (MuSK) antibody-positive MG...Our preliminary survey data suggests that despite efforts to create a set of international consensus guidelines, uncertainty persists in the neurology community surrounding MG management. We identify areas of agreement and disagreement that can help clinicians refine their own practice patterns and define the need for modified guidelines that highlight the side effect profiles, cost, and availability of therapies worldwide.
  • ||||||||||  MR16-1 / Roche
    Anti-IL-6 Receptor Antibody Prevents Muscle Weakness in Experimental Autoimmune Myasthenia Gravis Mouse Model (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4257;    
    We identify areas of agreement and disagreement that can help clinicians refine their own practice patterns and define the need for modified guidelines that highlight the side effect profiles, cost, and availability of therapies worldwide. Anti-IL-6R antibody prevented development of muscle weakness and reduced anti-AChR antibody production and deposition in AChR-immunized EAMG mice.
  • ||||||||||  Artificial Intelligence-based Ocular Motor Biomarkers for Myasthenia Gravis Diagnosis (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4256;    
    Anti-IL-6R antibody prevented development of muscle weakness and reduced anti-AChR antibody production and deposition in AChR-immunized EAMG mice. The preliminary results suggest that deep learning is well-suited to develop an automated myasthenia diagnostic tool from a larger dataset of OKN video recordings.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Patterns of Steroid Use and Outcomes in US Patients with Generalized Myasthenia Gravis (gMG) Receiving C5 Inhibitor Therapy (C5IT) (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4251;    
    C5ITs eculizumab and ravulizumab are approved gMG treatments that employ targeted inhibition of terminal complement activation to reduce neuromuscular junction damage. These medical claims data showed statistically significant reductions in prednisone ADD after 12-months' C5IT and gMG exacerbations and crises during 6 months of C5IT treatment compared with 6 months before C5IT initiation.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Fixed-cycle and Continuous Dosing of Intravenous Efgartigimod for Generalized Myasthenia Gravis: Study Design of ADAPT NXT (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4250;    
    P3
    These medical claims data showed statistically significant reductions in prednisone ADD after 12-months' C5IT and gMG exacerbations and crises during 6 months of C5IT treatment compared with 6 months before C5IT initiation. ADAPT NXT will provide important data on different treatment regimens, which will allow for further individualization of treatment with efgartigimod.
  • ||||||||||  gefurulimab (ALXN1720) / AstraZeneca
    Phase 1 Study of Gefurulimab Pharmacokinetics (PK) and Safety Following Delivery Via Autoinjector in Healthy Adults (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4244;    
    P3
    This study will examine the PK, PD, safety, tolerability, immunogenicity, and device performance of the gefurulimab autoinjector in healthy adults. Results may support use of the autoinjector with the goals of providing convenient administration and enhancing patient experience in individuals with gMG.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Effectiveness and Safety of Transitioning to Ravulizumab from Eculizumab in Patients with Generalized Myasthenia Gravis: Evidence from a Global Registry (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_4242;    
    Physicians also initiated a new cycle in some patients with MG-ADL or QMG score changes that did not meet the suggested threshold for new cycle initiation, indicating that physicians personalized treatment based on their understanding of individual patient needs. These initial results from a global registry represent clinical practice and suggest that transitioning from Q2W eculizumab to Q8W ravulizumab is safe and effective for patients with AChR+ gMG.
  • ||||||||||  clonazepam / Generic mfg.
    The "Drama Mask" Phenomenon in Craniofacial Dystonia (Colorado Convention Center | Exhibit Hall B-E) -  Mar 8, 2024 - Abstract #AAN2024AAN_3893;    
    Clonazepam had minimal impact...This case report shows that craniofacial dystonia can impact outward expression of emotions, even producing emotionally incongruent facial expressions. This "drama mask" phenomenon can be socially disabling, but is treatable with targeted botulinum toxin injections.