keyword
https://read.qxmd.com/read/38534400/musk-myasthenia-gravis-potential-pathomechanisms-and-treatment-directed-against-specific-targets
#1
REVIEW
Edyta Dziadkowiak, Dagmara Baczyńska, Marta Waliszewska-Prosół
Myasthenia gravis (MG) is an autoimmune disease in which autoantibodies target structures within the neuromuscular junction, affecting neuromuscular transmission. Muscle-specific tyrosine kinase receptor-associated MG (MuSK-MG) is a rare, often more severe, subtype of the disease with different pathogenesis and specific clinical features. It is characterized by a more severe clinical course, more frequent complications, and often inadequate response to treatment. Here, we review the current state of knowledge about potential pathomechanisms of the MuSK-MG and their therapeutic implications as well as ongoing research in this field, with reference to key points of immune-mediated processes involved in the background of myasthenia gravis...
March 21, 2024: Cells
https://read.qxmd.com/read/38534352/recombinant-acetylcholine-receptor-immunization-induces-a-robust-model-of-experimental-autoimmune-myasthenia-gravis-in-mice
#2
JOURNAL ARTICLE
Lukas Theissen, Christina B Schroeter, Niklas Huntemann, Saskia Räuber, Vera Dobelmann, Derya Cengiz, Alexander Herrmann, Kathrin Koch-Hölsken, Norbert Gerdes, Hao Hu, Philipp Mourikis, Amin Polzin, Malte Kelm, Hans-Peter Hartung, Sven G Meuth, Christopher Nelke, Tobias Ruck
Myasthenia gravis (MG) is a prototypical autoimmune disease of the neuromuscular junction (NMJ). The study of the underlying pathophysiology has provided novel insights into the interplay of autoantibodies and complement-mediated tissue damage. Experimental autoimmune myasthenia gravis (EAMG) emerged as a valuable animal model, designed to gain further insight and to test novel therapeutic approaches for MG. However, the availability of native acetylcholine receptor (AChR) protein is limited favouring the use of recombinant proteins...
March 14, 2024: Cells
https://read.qxmd.com/read/38533739/rozanolixizumab-a-new-therapy-in-the-treatment-of-myasthenia-gravis
#3
REVIEW
Emily M Hitt
OBJECTIVE: The aims of this article are to review the clinical aspects of rozanolixizumab, to describe clinical trial results that led to the drug's approval, and to examine the impact on patient care to aid clinical decision making. DATA SOURCES: A PubMed search was conducted using the terms Rystiggo ™, rozanolixizumab , rozanolixizumab therapy , and myasthenia gravis . The most recent prescribing information was also used for information relating to the drug and for identification of pertinent studies...
March 27, 2024: Annals of Pharmacotherapy
https://read.qxmd.com/read/38533199/study-on-the-clinical-and-electrophysiological-characteristics-of-nerve-function-in-myasthenia-gravis-patients-in-vietnam
#4
JOURNAL ARTICLE
Tho Kieu Anh Pham, Phuong Van Pham, Tung Dinh Le, Binh Thanh Nguyen, Trung Kien Nguyen, Tam Thai Thanh Tran, Sam Phan Hai Nguyen, Minh Van Le
BACKGROUND: In Vietnam, there is limited research on the role of nerve conduction in myasthenia gravis and its association with clinical features. OBJECTIVE: This study aims to describe the electrophysiological features in patients with myasthenia gravis. METHODS: This descriptive study was conducted from September 2019 to December 2021. The study included 33 myasthenia gravis patients who sought medical consultation or received inpatient treatment during this period...
2024: SAGE Open Medicine
https://read.qxmd.com/read/38532873/case-report-neurological-adverse-events-in-subject-with-myasthenia-gravis-after-pcsk9-inhibitor-administration
#5
Věra Adámková, Martina Vitásková, Jaroslav A Hubáček
BACKGROUND: Myasthenia gravis is a rare chronic autoimmune neuromuscular disorder mainly caused by autoantibodies to the nicotinic acetylcholine receptor. Cholesterol is an essential molecule that affects the distribution and proper functioning of this receptor. Several reports have described the potential worsening of myasthenia gravis in patients treated with statins. CASE PRESENTATION: The patient was an obese 72 years old man, past smoker, diagnosed with ischaemic heart disease, type 2 diabetes mellitus and lipid metabolism disorder...
2024: Frontiers in Cardiovascular Medicine
https://read.qxmd.com/read/38530530/the-prognostic-role-of-the-number-of-involved-structures-in-thymic-epithelial-tumors-results-from-the-ests-database
#6
JOURNAL ARTICLE
Marco Chiappetta, Filippo Lococo, Carolina Sassorossi, Clemens Aigner, Till Ploenes, Dirk Van Raemdonck, Cedric Vanluyten, Paul Van Schil, Apostolos C Agrafiotis, Francesco Guerrera, Paraskevas Lyberis, Monica Casiraghi, Lorenzo Spiaggiari, Charalambos Zisis, Christina Magou, Bernhard Moser, Jonas Bauer, Pascal Alexandre Thomas, Geoffrey Brioude, Stefano Passani, Zalan Zsanto, Isabella Sperduti, Stefano Margaritora
BACKGROUND: The role of the number of involved structures (NIS) in thymic epithelial tumors (TETs) has been investigated for inclusion in future staging systems, but large cohort results still are missing. This study aimed to analyze the prognostic role of NIS for patients included in the European Society of Thoracic Surgeons (ESTS) thymic database who underwent surgical resection. METHODS: Clinical and pathologic data of patients from the ESTS thymic database who underwent surgery for TET from January 2000 to July 2019 with infiltration of surrounding structures were reviewed and analyzed...
March 26, 2024: Annals of Surgical Oncology
https://read.qxmd.com/read/38529037/treatment-strategies-and-treatment-related-adverse-events-in-mg-according-to-the-age-of-onset
#7
JOURNAL ARTICLE
João Moura, Joana Fernandes, Maria João Lima, Ana Paula Sousa, Raquel Samões, Ana Martins Silva, Ernestina Santos
INTRODUCTION: Early-onset (EOMG) and late-onset (LOMG) are distinct groups of MG patients. It is unclear if treatment strategies and treatment-related adverse events may differ according to the age of MG onset. METHODS: This single-center retrospective study includes all MG patients followed at a tertiary center since 2007. We reviewed the electronic clinical records. RESULTS: In total, 212 patients were identified, 142 (67.0%) females, with a median disease duration of 10 years...
2024: Frontiers in Neurology
https://read.qxmd.com/read/38528163/real-world-experience-with-efgartigimod-in-patients-with-myasthenia-gravis
#8
JOURNAL ARTICLE
Lior Fuchs, Shahar Shelly, Ifat Vigiser, Hadar Kolb, Keren Regev, Yoel Schwartzmann, Adi Vaknin-Dembinsky, Amir Dori, Arnon Karni
Recommendations for the treatment of myasthenia gravis (MG) have been difficult to develop because of limited evidence from large randomized controlled trials. New drugs and treatment approaches have recently been shown to be effective in phase 3 studies in seropositive generalized (g) MG. One such drug is efgartigimod, a human-Fc-fragment of IgG1, with a high affinity for the endosomal FcRn. We conducted a multicenter study to evaluate the real-world clinical and safety effects of efgartigimod in 22 gMG patients...
March 25, 2024: Journal of Neurology
https://read.qxmd.com/read/38523857/co-existence-of-type-1-diabetes-mellitus-and-myasthenia-gravis-a%C3%A2-case-report-and-review-of-the-literature
#9
Sabitha Sasidharan Pillai, Kate Millington
BACKGROUND/OBJECTIVE: Type 1 diabetes (T1D) and myasthenia gravis (MG) are autoimmune conditions that rarely co-occur. Here, we report a child with MG who subsequently developed T1D. CASE REPORT: An 11-year-old girl with seropositive MG diagnosed at 4 years of age presented with muscle pain, cramps, and weight loss of 3.5 kg over 4 months. Her MG was in remission on daily pyridostigmine. She denied polyuria, polydipsia, recent illnesses, or other medications...
2024: AACE Clinical Case Reports
https://read.qxmd.com/read/38523508/promising-therapies-for-the-treatment-of-myasthenia-gravis
#10
REVIEW
Sanem Pinar Uysal, John A Morren
INTRODUCTION: Myasthenia gravis (MG) is an autoimmune condition targeting the neuromuscular junction, which manifests with neuromuscular symptoms of varying severity and significant morbidity. The mainstay of treatment in MG is mitigation of the immune cascade with steroids and non-steroidal immunosuppressive therapies. The therapeutic strategies in MG are transitioning from broad and indiscriminate immunosuppression to novel agents targeting key steps in MG pathogenesis, including T cell activation, B cell proliferation, complement activation, maintenance of pathogenic antibody production, and proinflammatory cytokine production...
March 25, 2024: Expert Opinion on Pharmacotherapy
https://read.qxmd.com/read/38523425/managing-suspected-myasthenia-gravis-and-myositis-induced-by-pembrolizumab-in-a-jehovah-s-witness-sarcoma-patient
#11
JOURNAL ARTICLE
Alexander Hutchinson, Danielle Eskens, Abigail Chan, Anika Bhargava, Ryan Bycroft
INTRODUCTION: Immune checkpoint inhibitors (ICIs) can cause a spectrum of adverse events known as immune-related adverse events (irAEs) that resemble autoimmune responses. Immune-mediated myasthenia gravis (MG) is a rare and serious neurologic adverse event that has been associated with ICIs requiring prompt treatment. In the Jehovah's Witness population, typical management of these adverse events may not be options, and alternative treatment choices would be needed. CASE REPORT: 73-year-old Jehovah's Witness patient with high-grade undifferentiated pleiomorphic sarcoma who developed immune-mediated MG approximately 4 weeks after initiation of pembrolizumab...
March 24, 2024: Journal of Oncology Pharmacy Practice
https://read.qxmd.com/read/38523419/expert-consensus-recommendations-for-improving-and-standardising-the-assessment-of-patients-with-generalised-myasthenia-gravis
#12
JOURNAL ARTICLE
Andreas Meisel, Francesco Saccà, Jennifer Spillane, John Vissing
BACKGROUND: Regular and consistent disease assessment could provide a clearer picture of burden in generalised myasthenia gravis (gMG) and improve patient care; however, the use of assessment tools in practice lacks standardisation. This modified Delphi approach was taken to review current evidence on assessment tool use in gMG and develop expert-derived consensus recommendations for good practice. METHODS: A European expert panel of 15 experienced gMG neurologists contributed to development of this consensus, four of whom formed a lead Sub-committee...
March 24, 2024: European Journal of Neurology
https://read.qxmd.com/read/38521921/integrative-multi-omics-analysis-identifies-genetically-supported-druggable-targets-and-immune-cell-specificity-for-myasthenia-gravis
#13
JOURNAL ARTICLE
Jiao Li, Fei Wang, Zhen Li, Jingjing Feng, Yi Men, Jinming Han, Jiangwei Xia, Chen Zhang, Yilai Han, Teng Chen, Yinan Zhao, Sirui Zhou, Yuwei Da, Guoliang Chai, Junwei Hao
BACKGROUND: Myasthenia gravis (MG) is a chronic autoimmune disorder characterized by fluctuating muscle weakness. Despite the availability of established therapies, the management of MG symptoms remains suboptimal, partially attributed to lack of efficacy or intolerable side-effects. Therefore, new effective drugs are warranted for treatment of MG. METHODS: By employing an analytical framework that combines Mendelian randomization (MR) and colocalization analysis, we estimate the causal effects of blood druggable expression quantitative trait loci (eQTLs) and protein quantitative trait loci (pQTLs) on the susceptibility of MG...
March 24, 2024: Journal of Translational Medicine
https://read.qxmd.com/read/38513158/pearls-oy-sters-thyroid-associated-ophthalmopathy-and-transient-neuromuscular-junction-disorder-due-to-graves-disease
#14
JOURNAL ARTICLE
Vasiliki Zouvelou, Elli M Petrou, Eleni Strataki, Vasiliki Vasileiou, Georgios Velonakis
The concomitant presentation of thyroid-associated ophthalmopathy (TAO) and ocular myasthenia gravis is well documented. In the course of Graves disease (GD), symptomatic transient neuromuscular junction disorder may occur due to the effect of thyroid hormones at the neuromuscular synapse. Diagnostic clues are the clinical and electrophysiologic remission synchronous with restoration of euthyroidism. Furthermore, the occurrence of thymic hyperplasia in GD poses further diagnostic and therapeutic considerations...
April 23, 2024: Neurology
https://read.qxmd.com/read/38508730/-a-case-of-myasthenia-gravis-with-coexistence-of-anti-acetylcholine-receptor-antibodies-and-anti-p-q-type-vgcc-antibodies
#15
JOURNAL ARTICLE
Yuki Takeda, Yoshikatsu Noda, Naohiko Seike, Hiroyuki Ishihara
A 79-year-old woman who presented ptosis and dysphagia were admitted to our hospital. Anti-acetylcholine receptor antibodies and anti-P/Q-type VGCC antibodies were both positive. Electrophysiological examination showed postsynaptic pattern which supported myasthenia gravis. She did not meet the diagnostic criteria for Lambert-Eaton myasthenic syndrome (LEMS). In cases which these antibodies coexist, careful electrophysiological evaluation is required for the diagnosis. In addition, although anti-P/Q-type VGCC antibodies have been specific to LEMS, patients with these antibodies represent various symptoms other than LEMS...
March 20, 2024: Rinshō Shinkeigaku, Clinical Neurology
https://read.qxmd.com/read/38507656/functional-signature-of-lrp4-antibodies-in-myasthenia-gravis
#16
JOURNAL ARTICLE
Omar Chuquisana, Frauke Stascheit, Christian W Keller, Maja Pučić-Baković, Anne-Marie Patenaude, Gordan Lauc, Socrates Tzartos, Heinz Wiendl, Nick Willcox, Andreas Meisel, Jan D Lünemann
BACKGROUND AND OBJECTIVES: Antibodies (Abs) specific for the low-density lipoprotein receptor-related protein 4 (LRP4) occur in up to 5% of patients with myasthenia gravis (MG). The objective of this study was to profile LRP4-Ab effector actions. METHODS: We evaluated the efficacy of LRP4-specific compared with AChR-specific IgG to induce Ab-dependent cellular phagocytosis (ADCP), Ab-dependent cellular cytotoxicity (ADCC), and Ab-dependent complement deposition (ADCD)...
May 2024: Neurology® Neuroimmunology & Neuroinflammation
https://read.qxmd.com/read/38507469/the-clc-1-chloride-channel-inhibitor-nmd670-improves-skeletal-muscle-function-in-rat-models-and-patients-with-myasthenia-gravis
#17
RANDOMIZED CONTROLLED TRIAL
Martin Skov, Titia Q Ruijs, Thomas S Grønnebæk, Marianne Skals, Anders Riisager, Jeppe Blichfeldt Winther, Kamilla Løhde Tordrup Dybdahl, Anders Findsen, Jeanette J Morgen, Nete Huus, Martin Broch-Lips, Ole B Nielsen, Catherine M K E de Cuba, Jules A A C Heuberger, Marieke L de Kam, Martijn Tannemaat, Jan J G M Verschuuren, Lars J S Knutsen, Nicholas M Kelly, Klaus G Jensen, William D Arnold, Arthur H Burghes, Claus Olesen, Jane Bold, Thomas K Petersen, Jorge A Quiroz, John Hutchison, Eva R Chin, Geert J Groeneveld, Thomas H Pedersen
Myasthenia gravis (MG) is a neuromuscular disease that results in compromised transmission of electrical signals at the neuromuscular junction (NMJ) from motor neurons to skeletal muscle fibers. As a result, patients with MG have reduced skeletal muscle function and present with symptoms of severe muscle weakness and fatigue. ClC-1 is a skeletal muscle specific chloride (Cl- ) ion channel that plays important roles in regulating neuromuscular transmission and muscle fiber excitability during intense exercise...
March 20, 2024: Science Translational Medicine
https://read.qxmd.com/read/38505076/anti-titin-antibodies-in-a-cohort-of-myasthenia-gravis-patients
#18
JOURNAL ARTICLE
João Moura, Ana Paula Sousa, Raquel Samões, Paula Carneiro, Esmeralda Neves, Ana Martins Silva, Ernestina Santos
BACKGROUND: Anti-titin antibodies have been previously associated with thymoma-associated myasthenia gravis (MG) and a more clinically severe form of MG. While currently only serving as a disease biomarker, its possible utility as an indicator of underlying thymus malignancy may be of value in clinical practice. METHODS: Data was retrospectively collected and analyzed from 2013 to 2022 using an institutional record of MG patients. Anti-titin antibodies were assessed using Line Blot immunoassay...
February 29, 2024: Journal of Thoracic Disease
https://read.qxmd.com/read/38499774/-myasthenia-gravis-gender-aspects-and-family-planning
#19
REVIEW
Sarah Hoffmann, Stefan Verlohren, Meret Herdick
BACKGROUND: There is evidence that gender-specific differences can influence the diagnostics, treatment and long-term disease course of myasthenia gravis (MG). In women the diagnosis is often made during childbearing age. OBJECTIVE: Gender-specific differences in MG and relevant aspects in routine clinical practice are presented. In addition, current studies on family planning, pregnancy and childbirth in MG are highlighted and treatment recommendations are derived...
March 18, 2024: Der Nervenarzt
https://read.qxmd.com/read/38494291/paraneoplastic-autoimmune-neurologic-disorders-associated-with-thymoma
#20
REVIEW
Raffaele Iorio, Vanda A Lennon
Thymoma is often associated with paraneoplastic neurologic diseases. Neural autoantibody testing is an important tool aiding diagnosis of thymoma and its autoimmune neurologic complications. Autoantibodies specific for muscle striational antigens and ion channels of the ligand-gated nicotinic acetylcholine receptor superfamily are the most prevalent biomarkers. The autoimmune neurologic disorders associating most commonly with thymoma are myasthenia gravis (MG), peripheral nerve hyperexcitability (neuromyotonia and Morvan syndrome), dysautonomia, and encephalitis...
2024: Handbook of Clinical Neurology
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