- |||||||||| Journal: What Is Myasthenia Gravis? (Pubmed Central) - Feb 7, 2024
This Review provides a comprehensive overview of the progress achieved in molecular therapies for MG associated with No abstract available
- |||||||||| Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
P3 data, P3 data: top line, Journal: Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. (Pubmed Central) - Feb 6, 2024 P3 Results of ADAPT+ corroborate the substantial clinical improvements seen with efgartigimod in ADAPT and support its long-term safety, tolerability, and efficacy, as well as an individualized dosing regimen for treatment of gMG. https://classic.clinicaltrials.gov/ct2/show/NCT03770403, NCT03770403.
- |||||||||| C139 - Neuromuscular Junction Disorders: Myasthenia Gravis, Ocular, and MuSK Myasthenia (Colorado Convention Center | Bluebird 2E-H) - Feb 5, 2024 - Abstract #AAN2024AAN_1276;
We also discuss and provide general recommendations for the evaluation and management of dysautonomia in these patients. Learning Objectives Participants should understand current methods of diagnosis of myasthenia gravis and be able to assess traditional and newer therapies used in myasthenia gravis.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Enrollment closed, Trial completion date, Trial primary completion date: Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) - Jan 31, 2024 P2, N=80, Active, not recruiting, To minimize risks for having affected child/children with autosomal recessive disorders, consanguineous couples must undergo genetic risk assessment, counseling and screening. Recruiting --> Active, not recruiting | Trial completion date: Jun 2033 --> Jan 2030 | Trial primary completion date: Jan 2028 --> Jan 2025
- |||||||||| Journal: Diagnosis and evaluation of elderly-onset myasthenia gravis: A case report. (Pubmed Central) - Jan 30, 2024
Recruiting --> Active, not recruiting | Trial completion date: Jun 2033 --> Jan 2030 | Trial primary completion date: Jan 2028 --> Jan 2025 A better understanding of the characteristics of elderly-onset myasthenia gravis may allow for relatively safe assessment of the condition and improve its diagnosis and treatment.
- |||||||||| Journal: Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease. (Pubmed Central) - Jan 30, 2024
Patient preference information can be collected using a variety of approaches, both qualitative and quantitative, tailored to fit the research needs of a study. The novel mixed-methods approach employed in this study efficiently captured patient preference data that were informative for exploratory research, internal decision making, and future research.
- |||||||||| Enrollment open, Trial completion date, Trial primary completion date: Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis: Multicenter Analysis in China (clinicaltrials.gov) - Jan 30, 2024
P=N/A, N=300, Recruiting, The novel mixed-methods approach employed in this study efficiently captured patient preference data that were informative for exploratory research, internal decision making, and future research. Not yet recruiting --> Recruiting | Trial completion date: Sep 2022 --> May 2028 | Trial primary completion date: Sep 2022 --> Feb 2028
- |||||||||| Review, Journal: Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future. (Pubmed Central) - Jan 29, 2024
The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.
- |||||||||| Journal: LRP4-related signalling pathways and their regulatory role in neurological diseases. (Pubmed Central) - Jan 29, 2024
The review also discusses the clinical and etiological roles of LRP4 in neurological illnesses, such as myasthenia gravis, Alzheimer's disease and epilepsy. In this review, we provide a theoretical foundation for the pathogenesis and therapeutic application of LRP4 in neurologic diseases.
- |||||||||| Journal: Depression: A Contributing Factor to the Clinical Course in Myasthenia Gravis Patients. (Pubmed Central) - Jan 27, 2024
Substantial reductions in MG-ADL and QMG scores were observed within each group after six months, highlighting the effectiveness of MG management. The findings suggest that addressing depressive symptoms in MG patients, in addition to standard MG management, can lead to improved clinical outcomes.
- |||||||||| Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Journal: The Clinical Course and Treatment of a Case of Refractory Systemic Juvenile Myasthenia Gravis Successfully Treated with Thymectomy. (Pubmed Central) - Jan 26, 2024 Despite undergoing various treatments, including steroids, tacrolimus, steroid pulse therapy, intravenous immunoglobulin, azathioprine (AZT), and rituximab, his symptoms did not improve. No abstract available
- |||||||||| Trial completion date, Trial primary completion date: VRMG: Vitaccess Real MG Registry (clinicaltrials.gov) - Jan 26, 2024
P=N/A, N=600, Not yet recruiting, No abstract available Trial completion date: Dec 2034 --> Mar 2034 | Trial primary completion date: Dec 2034 --> Mar 2034
- |||||||||| Remicade (infliximab) / J&J, Opdivo (nivolumab) / Ono Pharma, BMS, Yervoy (ipilimumab) / Ono Pharma, BMS
OF BROKEN MUSCLES AND BROKEN HEARTS: ICI-MEDIATED FATAL MYOSITIS AND MYOCARDITIS (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_7488; Myocarditis may occur concurrently with myositis or myasthenia gravis in approximately 30% patients. The risk of developing myocarditis was higher among patients receiving combination anti-PD-1 based therapies.
- |||||||||| THE IMPACT OF MYASTHENIA GRAVIS ON THE RISK OF COVID-19-RELATED MYOCARDITIS (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_5488;
Our findings suggest that autoantibodies in context of MG do not increase the risk of COVID-19-related MC. Our findings are limited by inherent bias associated with retrospective studies and the absence of adjustments for other chronic comorbidities.
- |||||||||| diltiazem / Generic mfg., heparin sodium injection / Generic mfg.
A CASE REPORT OF ATRIAL FIBRILLATION IN MUSCLE SPECIFIC TYROSINE KINASE MYASTHENIC CRISIS (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_3770; She was stabilized with fluid resuscitation and electrolyte repletion then rate control was achieved with metoprolol and diltiazem... Further clinical and basic studies are needed to fully understand the relationship between myasthenic crisis and atrial fibrillation.
- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
KEY TO THE HEART: AN IMMUNE CHECKPOINT INHIBITOR-ASSOCIATED CARDIAC AND NEUROLOGIC TOXICITY (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_3330; While rare, cases of ICI-induced myocarditis with myositis/myasthenia gravis overlap syndrome have been reported in recent years. Recognition of this overlap syndrome and prompt initiation of treatment may be the key in improving outcomes of this life-threatening condition.
- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
PEMBROLIZUMAB ASSOCIATED MYOCARDITIS AND ITS VARIED CLINICAL PRESENTATION (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_1337; Had this drug side effect been identified earlier, his subsequent decompensation may have been avoided. There should be a high index of suspicion for myocarditis in patients receiving immune checkpoint inhibitors and this case highlights some of the many ways it can present.
- |||||||||| Journal: Alteration of actin cytoskeletal organisation in fetal akinesia deformation sequence. (Pubmed Central) - Jan 24, 2024
By proximity ligation assays and bimolecular fluorescence complementation, we show that rapsyn and NUP88 localise nearby adhesion plaques and that they interact with the focal adhesion protein paxillin. Based on these findings we propose that a respective deficiency in rapsyn and NUP88 in FADS alters the regulation of actin dynamics at focal adhesions, and thereby may also plausibly dictate myofibril contraction in skeletal muscle of FADS individuals.
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