- |||||||||| [VIRTUAL] AAV Platform Vector Gene Therapy (PAVE-GT) for Congenital Myasthenic Syndomes () - Apr 30, 2021 - Abstract #ASGCT2021ASGCT_611;
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 established animal models in order 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 learnings from this fully available.
- |||||||||| Retrospective data, Journal: Prosthetic Reconstruction of Superior Vena Cava System for Thymic Tumor: A Retrospective Analysis of 22 Cases. (Pubmed Central) - Apr 30, 2021
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 learnings from this fully available. Our study, confirming data from existing literature, showed that the prosthetic reconstruction of the SVC system is a feasible additional procedure during resection of thymic tumor infiltrating the venous mediastinal axis, minimally increasing postoperative complications in experienced hands.
- |||||||||| Clinical, Journal: Confounding case of seromucinous hamartoma. (Pubmed Central) - Apr 30, 2021
Subsequent blood work demonstrated high titres of anti-acetylcholine receptor antibodies consistent with myasthenia gravis. The patient was started on pyridostigmine with improvement in his ocular cranial neuropathies.
- |||||||||| prednisolone / Generic mfg.
Journal, Causal relationship: Trauma-induced myasthenia gravis: coincidence or causal relationship? (Pubmed Central) - Apr 30, 2021 In this report, we describe the timing and progression of MG in our patient, and review the literature pertaining to the relationship between trauma and MG. The search for definitive evidence of causation may be impractical, but should not delay the recognition and management of a treatable condition.
- |||||||||| Journal, IO biomarker: Reduced plasmablast frequency is associated with seronegative myasthenia gravis. (Pubmed Central) - Apr 29, 2021
The search for definitive evidence of causation may be impractical, but should not delay the recognition and management of a treatable condition. Reduced plasmablast frequencies are strongly associated with a SN MG diagnosis and may be a useful diagnostic biomarker in the future.
- |||||||||| Journal: Self-Organizing 3D Human Trunk Neuromuscular Organoids. (Pubmed Central) - Apr 28, 2021
They contract and develop central pattern generator-like neuronal circuits. Finally, we successfully use NMOs to recapitulate key aspects of myasthenia gravis pathology, thus highlighting the significant potential of NMOs for modeling neuromuscular diseases in the future.
- |||||||||| Review, Journal: Neuromuscular Complications of Statin Therapy. (Pubmed Central) - Apr 28, 2021
In addition, statins can lead to unmasking or triggering of underlying muscle and neuromuscular junction disorders. This article identifies the risk factors and provides a review of neuromuscular side effects associated with statin use, their diagnosis and treatment.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Monoclonal Antibody-Based Therapies for Myasthenia Gravis. (Pubmed Central) - Apr 28, 2021 Thus far, only eculizumab, an antibody against C5, has reached the clinic. We review the present status of monoclonal antibody-based treatments for MG that have entered human testing and offer the promise to transform treatment of MG.
- |||||||||| Review, Journal, Checkpoint inhibition: Expert Perspective: Immune Checkpoint Inhibitors and Rheumatologic Complications. (Pubmed Central) - Apr 28, 2021
Corticosteroids and holding the ICI are usually required to treat myositis; some patients with severe myositis need intravenous immunoglobulin or plasmapheresis. Further research is needed to optimize treatment of irAEs that does not compromise the anti-tumor effect of ICIs.
- |||||||||| Journal: Congenital Ophthalmoplegia and Late-Onset Limb Weakness Caused by MUSK Mutations. (Pubmed Central) - Apr 27, 2021
He was misdiagnosed as mitochondrial myopathy because of paraclinic and histologic findings, but detailed clinical examination prompted us to reassess him with repetitive stimulation technique, demonstrating decremental response and suggesting myasthenic syndrome. A genetic study confirmed the clinical diagnosis allowing us to started treatment with excellent clinical response.
- |||||||||| Journal: Takotsubo Cardiomyopathy in Myasthenic Crisis. (Pubmed Central) - Apr 27, 2021
A genetic study confirmed the clinical diagnosis allowing us to started treatment with excellent clinical response. No abstract available
- |||||||||| Journal, Checkpoint inhibition: Development of carpal tunnel syndrome in association with checkpoint inhibitors. (Pubmed Central) - Apr 27, 2021
Subsequent correct therapeutic interventions resulted in remarkable recovery in functional status and prevented her from further aspiration in the long run. Given the prevalence of carpal tunnel syndrome in the general population, a high index of suspicion for carpal tunnel in patients receiving checkpoint inhibitors and prompt treatment with corticosteroids is essential.
- |||||||||| Clinical, Journal: Perioperative risk factors for occurrence of myasthenia gravis after thymectomy in patients with thymoma. (Pubmed Central) - Apr 27, 2021
Given the prevalence of carpal tunnel syndrome in the general population, a high index of suspicion for carpal tunnel in patients receiving checkpoint inhibitors and prompt treatment with corticosteroids is essential. This study revealed that incomplete tumour resection and thymomectomy were independent risk factors for PMG in thymoma patients with no previous history of myasthenia gravis.
- |||||||||| nipocalimab (M281) / J&J
[VIRTUAL] Vivacity-MG: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Nipocalimab Administered to Adults with Generalized Myastheni () - Apr 25, 2021 - Abstract #AAN2021AAN_3655; This condition is not only associated with significant morbidity and mortality, but also with unnecessary tests and procedures, iatrogenesis, prolonged hospitalizations and increased health costs. Nipocalimab was well-tolerated, safe, and efficacious in patients with gMG.
- |||||||||| Trial completion date, Trial primary completion date: SAA: Serum Auto-Antibodies in Neurological Diseases (clinicaltrials.gov) - Apr 22, 2021
P=N/A, N=120, Enrolling by invitation, Trial primary completion date: May 2021 --> Sep 2021 Trial completion date: Dec 2020 --> Mar 2031 | Trial primary completion date: Dec 2020 --> Mar 2031
- |||||||||| Opdivo (nivolumab) / Ono Pharma, BMS
Journal: Myasthenia gravis associated with nivolumab (Pubmed Central) - Apr 22, 2021 Trial completion date: Dec 2020 --> Mar 2031 | Trial primary completion date: Dec 2020 --> Mar 2031 No abstract available
- |||||||||| Review, Journal: Cholesterol in myasthenia gravis. (Pubmed Central) - Apr 21, 2021
This review analyzes some aspects of myasthenia gravis pathophysiology and associated postsynaptic membrane dysfunction, including dysregulation of cholesterol metabolism in the myocyte brought about by antibody-receptor interactions. In addition, given the extensive therapeutic use of statins as the typical cholesterol-lowering drugs, we discuss their effects on skeletal muscle and the possible implications for MG patients under chronic treatment with this type of compound.
- |||||||||| Review, Journal: Influence of cannabinoids upon nerve-evoked skeletal muscle contraction. (Pubmed Central) - Apr 20, 2021
Improved understanding of the distinct signaling actions of particular cannabinoid compounds and their receptor/transduction systems will help advance our understanding of the role of endocannabinoids in skeletal muscle physiology. Cannabinoids might also offer the potential to develop new pharmacotherapeutics to treat neuromuscular disorders that affect muscle strength.
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