- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
[VIRTUAL] Pembrolizumab Induced Myasthenia Gravis () - Mar 14, 2021 - Abstract #ATS2021ATS_2805;
She received five sessions of IVIG and was discharged on a prednisone taper and pyridostigmine, with complete resolution of her symptoms. This case highlights to need for close monitoring of patients on immune checkpoint inhibitor with unexplained respiratory failure.
- |||||||||| [VIRTUAL] Progressive Dyspnea and Ataxia- A Case of Coexisting Myasthenia Gravis and Pernicious Anemia () - Mar 14, 2021 - Abstract #ATS2021ATS_2790;
Notably, there is only one other published case report found through literature review. The diagnosis of coexisting pernicious anemia and myasthenia gravis in a patient with progressive dyspnea, ataxia, and weakness she the potential for overlap in both these autoimmune syndrome symptoms; and this demonstrates the reality that dyspnea is a complicated complaint that may have more than one cause.
- |||||||||| [VIRTUAL] Looking at Thymoma through the Aortopulmonary Window () - Mar 14, 2021 - Abstract #ATS2021ATS_2278;
A high index of suspicion is required prior to surgical resection even when outside of the anterior mediastinum. If thymoma is considered, evaluation for myasthenia gravis is required to prevent perioperative complications.
- |||||||||| Clinical, Journal: Surgical outcomes of 215 patients with thymic epithelial tumors: A single-center experience. (Pubmed Central) - Mar 12, 2021
What this study adds This study had a relatively large sample size of patients with thymic epithelial tumors in a single center. We evaluated not only the oncological prognosis, but also neurological outcomes after thymectomy, which was a more comprehensive assessment of surgical effect for thymic epithelial tumors.
- |||||||||| Journal: Affinity maturation is required for pathogenic monovalent IgG4 autoantibody development in myasthenia gravis. (Pubmed Central) - Mar 12, 2021
Crystal structures of two Fabs revealed how mutations acquired during affinity maturation may contribute to increased MuSK-binding affinity. These findings indicate that the autoantigen drives autoimmunity in MuSK MG through the accumulation of somatic mutations such that monovalent IgG4 Fab-arm-exchanged autoantibodies reach a high-affinity threshold required for pathogenic capacity.
- |||||||||| Trial completion, Enrollment change, Trial completion date, Trial primary completion date: Physical Activity and Fatigue in Myasthenia Gravis (clinicaltrials.gov) - Mar 10, 2021
P=N/A, N=779, Completed,
N=32 --> 0 | Trial completion date: Oct 2021 --> Jan 2021 | Not yet recruiting --> Withdrawn Recruiting --> Completed | N=278 --> 779 | Trial completion date: Oct 2021 --> Oct 2020 | Trial primary completion date: Apr 2021 --> Apr 2020
- |||||||||| Review, Journal: Autoantibody Specificities in Myasthenia Gravis; Implications for Improved Diagnostics and Therapeutics. (Pubmed Central) - Mar 9, 2021
We will herein review the advances made by us and others toward development of more sensitive detection methods and the identification of new antibody targets in MG, and discuss their significance in MG diagnosis and therapy. Overall, the development of novel autoantibody assays is aiding in the more accurate diagnosis and classification of MG patients, supporting the development of advanced therapeutics and ultimately the improvement of disease management and patient quality of life.
- |||||||||| Review, Journal: New Approaches to Targeting B Cells for Myasthenia Gravis Therapy. (Pubmed Central) - Mar 9, 2021
This review article provides an overview of B cell-targeted treatments for MG, including those already available and those still in preclinical and clinical development. We also discuss the potential benefits as well as the shortcomings of these approaches to development of new therapies for MG and future directions in the field.
- |||||||||| prednisone / Generic mfg.
Journal, IO biomarker: miR-146a in Myasthenia Gravis Thymus Bridges Innate Immunity With Autoimmunity and Is Linked to Therapeutic Effects of Corticosteroids. (Pubmed Central) - Mar 9, 2021
Indeed, an effect of prednisone on miR-146a expression was demonstrated in vitro on peripheral blood cells...Our overall findings strongly suggest that defective miR-146a expression could contribute to persistent TLR activation, lack of inflammation resolution, and hyperplastic changes in MG thymuses, thus linking TLR-mediated innate immunity to B-cell-mediated autoimmunity. Furthermore, they unraveled a new mechanism of action of corticosteroids in inducing control of autoimmunity in MG via miR-146a.
- |||||||||| Review, Journal: A tale of autoimmunity: thymoma, thymectomy, and systemic lupus erythematosus. (Pubmed Central) - Mar 9, 2021
Rare cases of post-thymectomy autoimmune related diseases, including systemic lupus erythematosus and pure red cell aplasia, have been reported in the literature. Here, we present the case of a 65-year-old male who developed systemic lupus erythematosus 2 years after thymectomy in the setting of thymoma-associated pure red cell aplasia.
- |||||||||| Tectin (tetrodotoxin) / WEX
Clinical, Review, Journal: Foodborne Botulism: Clinical Diagnosis and Medical Treatment. (Pubmed Central) - Mar 9, 2021
Foodborne botulism intoxication is often underdiagnosed; the initial symptoms can be confused with more common clinical conditions (i.e., stroke, myasthenia gravis, Guillain-Barré syndrome-Miller-Fisher variant, Eaton-Lambert syndrome, tick paralysis and shellfish or tetrodotoxin poisoning). The treatment includes procedures for decontamination, antidote administration and, when required, support of respiratory function; few differences are related to the different way of exposure.
- |||||||||| Clinical, Journal, Checkpoint inhibition: Inflammatory myositis in cancer patients receiving immune checkpoint inhibitors. (Pubmed Central) - Mar 9, 2021
His personal and firsthand experience expands on our increasing knowledge of immune checkpoint inhibitor (ICI)-induced myositis and ICI-myasthenia gravis (MG) from a clinical and laboratory perspective. We agree with observations made in Japanese patients, that accompanying MG in these ICI-myositis patients is difficult to tease out clinically, by electrodiagnostic evaluation (except via single-fiber electromyography-SFEMG) and laboratory investigations.
- |||||||||| Clinical, Journal: Neonatal Thymectomy in Children - Accelerating the Immunological Clock? (Pubmed Central) - Mar 6, 2021
In conclusion, infant thymectomy may increase future risk of infection or autoimmunity with premature immunosenescence, mediated through disruption of central and peripheral tolerance mechanisms, initiated by early cessation or diminution of thymic output. Ideally, some thymic tissue should be preserved at time of surgery.
- |||||||||| Review, Journal: Neonatal Fc receptor in human immunity: Function and role in therapeutic intervention. (Pubmed Central) - Mar 6, 2021
The effectiveness of FcRn inhibitors in autoimmune diseases, including myasthenia gravis and immune thrombocytopenia, provides further evidence that IgG is a primary driver in these autoantibody-mediated diseases. We describe the role of FcRn in human biology, including insights that clinical testing of FcRn inhibitors have provided into FcRn biology and autoimmune disease mechanisms, allowing fact-based speculation on their therapeutic potential.
- |||||||||| Journal: Neurologic complications of myocarditis. (Pubmed Central) - Mar 5, 2021
The emerging science will be discussed. Nuances of brain death (BD) determination in patients receiving venoarterial ECMO for heart failure refractory to standard medical therapies will be discussed.
- |||||||||| Journal: New recessive mutations in SYT2 causing severe presynaptic congenital myasthenic syndromes. (Pubmed Central) - Mar 5, 2021
In-frame skipping of exon 4 that encodes part of the C2A calcium-binding domain of SYT2 is associated with a loss-of-function effect resulting in a decrease of neurotransmitter release and severe pre- and postsynaptic NMJ defects. This study identifies new homozygous recessive SYT2 mutations as the underlying cause of severe and early presynaptic form of CMS expanding the genetic spectrum of recessive SYT2-related CMS associated with defects in neurotransmitter release.
- |||||||||| [VIRTUAL] Biallelic loss of function variants in SYT2 cause a treatable congenital onset presynaptic myasthenic syndrome () - Mar 4, 2021 - Abstract #MDA2021MDA_98;
In AChR+ gMG and AQP4+ NMOSD, infection rates were similar in eculizumab and placebo groups, regardless of concomitant ISTs, and were consistent with eculizumab’s established safety profile. This series further establishes SYT2 as a CMS-disease gene and expands its clinical and genetic spectrum to include recessive loss-of-function variants, manifesting as a severe congenital onset presynaptic CMS with potential treatment implications.
- |||||||||| [VIRTUAL] AAV Platform Vector Gene Therapy (PaVe-GT) for DOK7 Congenital Myasthenic Syndromes () - Mar 4, 2021 - Abstract #MDA2021MDA_87;
DOK7 is a regulator of acetylcholine receptor assembly necessary for postsynaptic NMJ formation. We have begun to study the use of self-complementary AAV and various vector/expression constructs to target the NMJ’s subsynaptic nuclei that will then be used to treat a Dok7 mouse model to determine if gene therapy is successful at restoring NMJ structure and muscle function.
- |||||||||| [VIRTUAL] AAV Platform Vector Gene Therapy (PaVe-GT) for COLQ Congenital Myasthenic Syndromes () - Mar 4, 2021 - Abstract #MDA2021MDA_86;
After determination of the most effective NMJ-targeting construct, we will deliver human codon-optimized COLQ transgene in preclinical Colq knockout mouse model to determine if the gene therapy is successful at restoring NMJ structure and muscle function. We thus plan to demonstrate this platform-based gene therapy approach from conception, preclinical optimization, production, toxicology studies, regulatory filings and interactions, all the way to a clinical trial and make all learning from this fully available.
- |||||||||| [VIRTUAL] Industry Forum Lunch Presented by Alexion Pharmaceuticals () - Mar 4, 2021 - Abstract #MDA2021MDA_7;
We thus plan to demonstrate this platform-based gene therapy approach from conception, preclinical optimization, production, toxicology studies, regulatory filings and interactions, all the way to a clinical trial and make all learning from this fully available. A Treatment for Adult Patients With Anti-Acetylcholine Receptor (AChR) Antibody-Positive Generalized Myasthenia Gravis (gMG)
- |||||||||| Journal: Henry R. Viets, MD, and the history of myasthenia gravis. (Pubmed Central) - Mar 3, 2021
He was the President of the American Association for the History of Medicine and curator of the Boston Medical Library that eventually joined with the Harvard Medical School Library. Viets served on the Editorial Board of the New England Journal of Medicine for 40 years.
- |||||||||| mezagitamab (TAK-079) / Takeda
Trial completion date, Trial primary completion date: A Study of TAK-079 in People With Generalized Myasthenia Gravis (clinicaltrials.gov) - Mar 3, 2021
P2, N=36, Recruiting,
Viets served on the Editorial Board of the New England Journal of Medicine for 40 years. Trial completion date: May 2021 --> Jun 2022 | Trial primary completion date: Feb 2021 --> Apr 2022
- |||||||||| pyridostigmine Bromide / Generic mfg.
Retrospective data, Review, Journal: Assessment of the risks of a myasthenic crisis after thymectomy in patients with myasthenia gravis: a systematic review and meta-analysis of 25 studies. (Pubmed Central) - Feb 27, 2021
These results implied that QJJLD may provide a potential therapeutic strategy through promoting mitochondrial biogenesis to alleviate MG via activating the AMPK/PGC-1α signaling pathway. The independent risk factors for MCAT were a preoperative history of MC, preoperative bulbar symptoms, preoperative MG Osserman stage, preoperative dosage of pyridostigmine bromide, preoperative serum AchR-Ab level, lung function, major postoperative complications, disease duration before thymectomy, blood loss, thoracotomy, postoperative lung infection, thymoma, and WHO classification.
- |||||||||| Clinical, Journal: Vitamin D Receptor Polymorphism and Myasthenia Gravis in Chinese Han Population. (Pubmed Central) - Feb 27, 2021
In the analysis of subgroups with a comprehensive classification, the frequencies of alleles and genotypes in rs731236 showed significant differences between adult non-thymoma AChRAb negative MG subgroup and the control group, as well as the adult non-thymoma AChRAb positive MG group. In the Chinese Han population, rs731236 was found to be possibly associated with adult non-thymoma AChRAb negative MG patients, although this needs further confirmation.
- |||||||||| Clinical, Observational data, Journal: Knowledge and Perceptions of the COVID-19 Pandemic Among Patients with Myasthenia Gravis. (Pubmed Central) - Feb 26, 2021
Health care providers, the government, and patient organizations play a critical role in communicating with the MG patient community. Use of targeted messaging strategies by these groups to convey accurate information may increase effectiveness and lead to more informed patients with reduced anxiety.
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