Myasthenia Gravis
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  • ||||||||||  Clinical, Journal:  Robotic thymectomy in thymic tumors: a multicenter, nation-wide study. (Pubmed Central) -  May 17, 2024   
    We found that circFRMD4, circPIGB and circNUP214 can be considered as valuable potential novel biomarkers for AchR Through the largest collection of robotic thymectomy for thymic epithelial tumours we demonstrated that robot-enhanced thoracoscopic thymectomy is a technically sound and safe procedure with a low complication rate and optimal oncological outcomes.
  • ||||||||||  Review, Journal:  FcRn Inhibitor Therapies in Neurologic Diseases. (Pubmed Central) -  May 16, 2024   
    Additionally, some of these agents may cause transient hypoalbuminemia and hypercholesterolemia notably reported with batoclimab and nipocalimab. In this review, we discuss the available clinical data for FcRN inhibitor agents in treating different neurological autoimmune diseases.
  • ||||||||||  Review, Journal:  Thymic carcinoma presenting with overlap polyarthritis and myositis: A rare paraneoplastic syndrome in childhood. (Pubmed Central) -  May 15, 2024   
    Herein, we report a case of thymic carcinoma mimicking the symptoms of a connective tissue disease with symmetrical polyarthritis accompanying myositis, fever, weight loss, and malaise in a 15-year-old male patient. To our knowledge, this is the first case pediatric thymic carcinoma accompany with severe polyarthritis and myopathy, thus we have reviewed the current literature regarding the cases of thymic malignancies coexisting with paraneoplastic syndromes in children.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Journal, Real-world evidence, Real-world:  Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis. (Pubmed Central) -  May 15, 2024   
    To our knowledge, this is the first case pediatric thymic carcinoma accompany with severe polyarthritis and myopathy, thus we have reviewed the current literature regarding the cases of thymic malignancies coexisting with paraneoplastic syndromes in children. Eculizumab and switching to ravulizumab showed to be effective for refractory AChR+ gMG patients in clinical settings.
  • ||||||||||  Empaveli (pegcetacoplan SC) / Apellis, SOBI
    EPIDEMIOLOGY AND REAL-WORLD TREATMENT MANAGEMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA IN FRANCE (Poster Area (Hall 7)) -  May 15, 2024 - Abstract #EHA2024EHA_3722;    
    More than 700 PNH patients were identified in 2022, with 100 newly diagnosed patients, leading to aprevalence of 1 case per 100,000 persons in France. This study brings updated data on the currentepidemiology in France and management of PNH which has a high treatment burden due to frequentinfusions.
  • ||||||||||  Review, Journal:  Lambert-Eaton Myasthenic Syndrome (Pubmed Central) -  May 14, 2024   
    Most patients benefit from 3,4-diaminopyridine administration for symptomatic treatment. A treatment algorithm is established by the clinical practice guidelines 2022.
  • ||||||||||  Review, Journal:  Muscle-Specific Receptor Tyrosine Kinase Antibody Positive Myasthenia Gravis (Pubmed Central) -  May 14, 2024   
    The aforementioned types of MG are indistinguishable based on clinical symptoms and electrophysiological tests, and measurement of MuSK antibodies is essential for diagnosis. Thymectomy and complement inhibitors are not indicated for treatment, and acetylcholinesterase inhibitors, steroids, immunosuppressants, plasma exchange, intravenous immunoglobulin therapy, and neonatal Fc receptor inhibitors are used.
  • ||||||||||  Review, Journal:  AChR Antibody-Positive Myasthenia Gravis (Pubmed Central) -  May 14, 2024   
    Downstream molecules include AChR antibodies that activate complement pathways, resulting in the destruction of motor endplates. We further introduce newly-developed molecular targeted drugs for the treatment of MG that aims to secure patients' health-related quality of life.
  • ||||||||||  Review, Journal, Adverse events, IO biomarker:  Peripheral Neuropathy and Muscle Disorders as Immune-Related Adverse Events (Pubmed Central) -  May 14, 2024   
    The administration of ICI in patients with small cell lung cancer increases the risk of LEMS. The distinction between LEMS is an irAE or a manifestation of paraneoplastic neurological syndrome is unclear as both conditions share common immunological mechanisms.
  • ||||||||||  Journal:  Innovative Therapeutic Approaches in Congenital Myasthenic Syndromes. (Pubmed Central) -  May 13, 2024   
    These therapies can greatly modify the prognosis of patients suffering from this orphan disease. This study provides Class IV evidence that for patients with congenital myasthenic syndromes, some innovative treatments are effective.
  • ||||||||||  Retrospective data, Review, Journal, Orphan drug:  Trends from two decades of orphan designations in paediatric rare neuromuscular diseases. (Pubmed Central) -  May 11, 2024   
    The large majority of PERAN diseases do not have an authorised medicine with only 6 OMP currently authorised for Spinal muscular atrophy (3); DMD (1) and Myasthenia gravis (2). One in five products have inactive or discontinued regulatory development but clinical trials are ongoing for the vast majority of PERAN diseases, and more than half are in the final stage of clinical research with significantly more products with medical plausibility based in clinical data reaching advanced stages in clinical development.
  • ||||||||||  Review, Journal:  Myasthenia gravis with Castleman disease: A case report with review of literature. (Pubmed Central) -  May 10, 2024   
    Here, we take the opportunity to describe a case of myasthenia gravis with Castleman disease simulating thymoma clinically and radiologically along with review of literature of this rare association. Patient developed myasthenic crisis in the immediate post-operative period which is more common in myasthenia gravis with Castleman disease rather than myasthenia gravis with thymoma.
  • ||||||||||  Trial completion, Enrollment change, Trial completion date, Trial primary completion date:  Clinical Trial to Improve the Magnetic Levator Prosthesis (clinicaltrials.gov) -  May 10, 2024   
    P=N/A,  N=17, Completed, 
    Our cohort of adult and pediatric patients expands the phenotype of DOK7 CMS and shows the importance of correct and early diagnosis. Active, not recruiting --> Completed | N=30 --> 17 | Trial completion date: Jul 2023 --> Dec 2023 | Trial primary completion date: Jul 2023 --> Dec 2023
  • ||||||||||  Enrollment closed, Trial completion date, Trial primary completion date:  Clinical Trial Comparing Two Non-Surgical Treatments for Severe Blepharoptosis (clinicaltrials.gov) -  May 9, 2024   
    P=N/A,  N=50, Active, not recruiting, 
    This study summarized the best evidence on the prevention and management of postoperative MC and provided to clinical staffs evidence-based clinical approaches to help reduce the incidence of this complication. Recruiting --> Active, not recruiting | Trial completion date: Jul 2024 --> Dec 2024 | Trial primary completion date: May 2023 --> Dec 2024
  • ||||||||||  Application of 68Ga-Pentixafor in evaluation of thymoma: a prospective study (801B (Convention Center); In-Person) -  May 8, 2024 - Abstract #SNMMI2024SNMMI_2264;    
    The preliminary study indicates the diagnostic utility of 68Ga-Pentixafor in thymoma and the differential diagnostic ability of 68Ga-Pentixafor in thymoma and thymic cyst. Further investigation of the differences between thymoma subtypes using 68Ga-Pentixafor in larger clinical trials is warranted.
  • ||||||||||  vemircopan (ALXN2050) / AstraZeneca
    Trial completion date, Trial termination:  ALXN2050-MG-201: Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) -  May 6, 2024   
    P2,  N=70, Terminated, 
    For conclusive evidences, more prospective randomized controlled trials will be needed in the future. Trial completion date: Dec 2025 --> Apr 2024 | Active, not recruiting --> Terminated; Sponsor decision
  • ||||||||||  Trial completion:  Acupuncture in Myasthenia Gravis (AcuMG) (clinicaltrials.gov) -  May 5, 2024   
    P=N/A,  N=24, Completed, 
    Trial completion date: Dec 2025 --> Apr 2024 | Active, not recruiting --> Terminated; Sponsor decision Active, not recruiting --> Completed
  • ||||||||||  Clinical, Retrospective data, Review, Journal:  Acupuncture and moxibustion treatment for myasthenia gravis: A systematic review and meta-analysis. (Pubmed Central) -  May 3, 2024   
    Some solid-phase assays (such as the indirect ELISA applied here), as well as CBA titration, offer reliable options to estimate anti-AChR AAb levels after confirming positivity by the CBA.?. Acupuncture and moxibustion has a good effect on MG, which is better than conventional Western medicine in improving the total clinical effective rate and absolute clinical score.
  • ||||||||||  Review, Journal:  Exploring the depths of IgG4: insights into autoimmunity and novel treatments. (Pubmed Central) -  May 3, 2024   
    Furthermore, we will examine potential therapeutic strategies for these conditions, with a special focus on leveraging insights gained from anti-CD20-mediated BCDT. Through this analysis, we aim to enhance our understanding of the pathogenesis of IgG4-mediated diseases and identify promising possibilities for targeted therapeutic intervention.