Myasthenia Gravis
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  • ||||||||||  azathioprine orodispersible / generics
    Journal:  Management of myasthenia gravis during pregnancy. (Pubmed Central) -  Jun 19, 2019   
    The key to successful outcome during pregnancy in myasthenic women lies in multidisciplinary care involving obstetricians, neurologists, anesthetist as well as neonatologist. In this review, we discuss various therapeutic options available for the management of MG during pregnancy and provide recommendations based on the current best evidence.
  • ||||||||||  Zilbrysq (zilucoplan) / UCB
    Trial completion date:  Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Jun 17, 2019   
    P2,  N=44, Active, not recruiting, 
    In this review, we discuss various therapeutic options available for the management of MG during pregnancy and provide recommendations based on the current best evidence. Trial completion date: Apr 2019 --> Apr 2020
  • ||||||||||  Journal:  Neuromyelitis optica spectrum disorder presenting with concurrent autoimmune diseases. (Pubmed Central) -  Jun 15, 2019   
    Our second patient had seronegative NMOSD with concurrent acetylcholine receptor antibody-positive MG. Practitioners should be aware of the potential for patients to present with a combination of NMOSD and one or more concurrent autoimmune diseases to ensure timely diagnosis and appropriate treatment.
  • ||||||||||  Journal:  Thoracoscopic thymectomy for juvenile myasthenia gravis. (Pubmed Central) -  Jun 15, 2019   
    Thoracoscopic thymectomy is a safe treatment for juvenile myasthenia gravis in pediatric patients over a wide range of ages, body masses, and symptoms. Our experience adds evidence that pediatric patients likely benefit from thymectomy with improved clinical status and reduced medications.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharmaceuticals
    Clinical, P3 data, Journal:  Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS. (Pubmed Central) -  Jun 15, 2019   
    Our experience adds evidence that pediatric patients likely benefit from thymectomy with improved clinical status and reduced medications. This phase 3 randomized, double-blind, placebo-controlled withdrawal trial in adults with LEMS provided class I evidence of efficacy of amifampridine phosphate as symptomatic treatment in LEMS.
  • ||||||||||  Review, Journal:  Lambert-Eaton Myasthenic syndrome: early diagnosis is key. (Pubmed Central) -  Jun 14, 2019   
    In clinically affected patients without cancer and after cancer treatment, symptomatic treatment with 3,4-diaminopyridine or immunosuppressive agents can significantly improve neurologic symptoms and the quality of life. We present a detailed review of LEMS with special emphasis on the pathophysiological mechanisms, clinical manifestation and diagnostic procedure.
  • ||||||||||  cemdisiran (ALN-CC5) / Alnylam
    Journal:  Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis. (Pubmed Central) -  Jun 14, 2019   
    Improvement in disease activity scores and NMJ pathology was observed at intermediate levels of complement activity inhibition, suggesting that complete ablation of complement activity may not be required for efficacy in MG. The pre-clinical studies of ALN-CC5 and efficacy of C5 silencing in rat models of MG support further clinical development of ALN-CC5 as a potential therapeutic for the treatment of MG and other complement-mediated disorders.
  • ||||||||||  Clinical, Journal:  Elevated expression of interleukin-33 in myasthenia gravis patients. (Pubmed Central) -  Jun 9, 2019   
    Increased serum IL-33 levels positively correlated with the upregulation of IL-17A levels and the quantitative myasthenia gravis (QMG) score in MG patients. Our findings indicate that IL-33 plays a potent immuno- enhancing role in the pathogenesis of MG by downregulating TSLP, consequently affecting the development of Th17 cells in MG.
  • ||||||||||  Journal:  Suppression of CHRN endocytosis by carbonic anhydrase CAR3 in the pathogenesis of myasthenia gravis. (Pubmed Central) -  Jun 7, 2019   
    CAR3 exerts this effect by suppressing chaperone-assisted selective autophagy via interaction with BAG3 (BCL2-associated athanogene 3) and by dampening endoplasmic reticulum stress. Collectively, our study illustrates that skeletal muscle cell CAR3 is critical for CHRN homeostasis in the neuromuscular junction, and its deficiency leads to accelerated degradation of CHRN and development of myasthenia gravis, potentially revealing a novel therapeutic approach for this disorder.
  • ||||||||||  Rituxan (rituximab) / Roche, Biogen
    Journal:  Rituximab in Treatment-Refractory Myasthenia Gravis. (Pubmed Central) -  Jun 5, 2019   
    Collectively, our study illustrates that skeletal muscle cell CAR3 is critical for CHRN homeostasis in the neuromuscular junction, and its deficiency leads to accelerated degradation of CHRN and development of myasthenia gravis, potentially revealing a novel therapeutic approach for this disorder. No abstract available
  • ||||||||||  Review, Journal:  Autoimmune Channelopathies at Neuromuscular Junction. (Pubmed Central) -  Jun 4, 2019   
    Although these channelopathies share some common features, they differ for clinical features, antibodies profile, neurophysiological features, and treatments. The purpose of this review is to give a comprehensive insight on recent advances in autoimmune channelopathies at the neuromuscular junction.
  • ||||||||||  Biomarker, Review, Journal:  Free light chains and autoimmunity. (Pubmed Central) -  Jun 3, 2019   
    This review deals with the use of polyclonal FLCs for identifying severity and monitoring outcome after treatment in some autoimmune diseases, namely systemic lupus erythematosus, myasthenia gravis, systemic sclerosis, rheumatoid arthritis and Sjögren's syndrome, as supported by the fact that levels of FLCs correlate with both B cell activation markers and other specific markers of disease activity. In a near future, following the evidence shown, FLCs might probably work as early prognostic markers of severity and also as indicators of response to treatment or early assessment of relapse in selected autoimmune diseases.
  • ||||||||||  Trial initiation date:  Can Interval Walking Influence on Fatigue in the Danish Cohort of Myasthenia Gravis Patients (clinicaltrials.gov) -  May 29, 2019   
    P=N/A,  N=32, Not yet recruiting, 
    We report on four consecutive patients with Parkinson's disease, in whom anti-acetylcholine receptor (AChR) antibody positive bulbar myasthenia gravis (MG) turned out to be responsible for progressive dysphagia. Initiation date: Apr 2019 --> Aug 2019
  • ||||||||||  Biomarker, Journal:  Serial stimulated jitter analysis in juvenile myasthenia gravis. (Pubmed Central) -  May 26, 2019   
    Serum interleukin 15 levels in patients with seropositive myasthenia gravis do not correlate with disease severity. Stimulated jitter values are sensitive biomarkers of disease activity in JMG.
  • ||||||||||  Clinical, Journal:  Global pathway view analysis of microRNA clusters in myasthenia gravis. (Pubmed Central) -  May 26, 2019   
    Additionally, the most significant pathways of the miRNA clusters were identified to be enriched on chromosomes 9, 19 and 22, characterized by immunity, infection and carcinoma, suggesting that the mechanism of MG may be associated with certain abnormalities of miRNA clusters on chromosomes 9, 19 and 22. The present study provides novel insight into a global pathway view of miRNA clusters in the pathogenesis of MG.
  • ||||||||||  NN1213 / Novo Nordisk
    Biomarker, Enrollment closed, Enrollment change:  Biomarkers in Neural Disorders (clinicaltrials.gov) -  May 24, 2019   
    P=N/A,  N=54, Active, not recruiting, 
    We have included this surgical procedure in our thoracic surgery residency program and recommend a learning curve program of 10 to 12 procedures during residency. Enrolling by invitation --> Active, not recruiting | N=440 --> 54
  • ||||||||||  prednisone / generics
    Review, Journal:  A Neurologist's Perspective on Thymectomy for Myasthenia Gravis: Current Perspective and Future Trials. (Pubmed Central) -  May 24, 2019   
    The first randomized blinded study of thymectomy in nonthymomatous myasthenia gravis was designed to answer 3 questions: does the combination of prednisone and removal of the thymus gland via extended transsternal thymectomy after 3 years compared with an identical dosing protocol of prednisone alone (1) lead to better disease status for generalized MG patients with antiacetylcholine receptor antibodies, (2) reduce their prednisone requirements, and/or (3) reduce the side-effect burden from medications used to treat the disease? The study demonstrated that thymectomy confers these benefits for patients and sets the stage for inquiries into the benefits of less-invasive approaches to thymic resection.
  • ||||||||||  Review, Journal:  History of Thymectomy for Myasthenia Gravis. (Pubmed Central) -  May 24, 2019   
    In the early 1900s, chance observations of improved symptoms in several myasthenic patients undergoing thyroidectomy for goiters with concomitant resection of the adjacent thymus gland, first suggested a possible association between the thymus and myasthenia gravis. With the remarkable progress made in the understanding and treatment of myasthenia and in the anesthetic, surgical, and postoperative management of patients undergoing thoracic procedures, the initial high morbidity and mortality associated with thymectomy have been all but eliminated, and thymectomy is frequently incorporated into the long-term management strategy of this disease.
  • ||||||||||  Review, Journal:  Surgical Approaches to Myasthenia Gravis: Perspective of Anatomy and Radicality in Surgery. (Pubmed Central) -  May 24, 2019   
    Surgical techniques include extended transsternal approach, which might be combined with separate transcervical incision, bilateral videothoracoscopic (VATS) approach combined with transcervical incision, and extended subxiphoid approach, either uniportal or combined with bilateral VATS. Anatomic aspects of maximally extensive thymectomy are described in detail.