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Design of and Early Insights From a Generalized Myasthenia Gravis Patient Engagement Research Council.
Health Sci Rep
December 2024
Janssen Scientific Affairs, LLC Titusville New Jersey USA.
Background And Aims: An exploratory focus group study was conducted to better understand the needs of patients living with generalized myasthenia gravis (gMG).
Methods: US-based adults with gMG and caregivers of patients with gMG participated in a Patient Engagement Research Council between August 2022 and January 2023. The study consisted of a 15-min prework survey, followed by virtual focus groups facilitated using a semi-structured interview guide.
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Front Neurol
October 2024
Department of Neurology, University of California, Irvine, Irvine, CA, United States.
Enzyme replacement therapy (ERT) is the only approved disease-modifying treatment modality for Pompe disease, a rare, inherited metabolic disorder caused by a deficiency in the acid -glucosidase (GAA) enzyme that catabolizes lysosomal glycogen. First-generation recombinant human GAA (rhGAA) ERT (alglucosidase alfa) can slow the progressive muscle degeneration characteristic of the disease. Still, most patients experience diminished efficacy over time, possibly because of poor uptake into target tissues.
View Article and Find Full Text PDFAssociation Between the Immune Checkpoint Inhibitor Durvalumab and Myasthenia Gravis: A Comprehensive Review.
Cureus
September 2024
Internal Medicine, Dayanand Medical College and Hospital, Ludhiana, IND.
Immune checkpoint inhibitors (ICIs), including Imfinzi (durvalumab), have revolutionized cancer treatment by stimulating the body's immune system to target cancerous cells. Although pharmaceuticals offer therapeutic benefits, several drugs have been associated with immune-related adverse events (irAEs), including the uncommon but serious condition known as myasthenia gravis (MG). This review synthesizes data from pertinent research to offer a thorough evaluation of the literature on the underlying mechanisms, clinical manifestations, and therapeutic approaches for durvalumab-induced MG.
View Article and Find Full Text PDFEfficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study.
Ther Adv Neurol Disord
September 2024
Department of Neurology, University Health Network, Toronto, ON, Canada.
Article Synopsis
- The study investigates the effectiveness and safety of rozanolixizumab in treating patients with muscle-specific tyrosine kinase (MuSK) autoantibody-positive generalized myasthenia gravis (gMG), which is a severe form of the condition.
- Conducted as a randomized, double-blind, placebo-controlled phase III trial, it involved participants aged 18 and older who were randomly assigned to receive either rozanolixizumab (at two different dosages) or a placebo over 6 weeks.
- Results showed significant improvements in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scores for both doses of rozanolixizumab compared to placebo, with manageable side effects reported;
[Differential diagnosis of vertical strabismus in the elderly].
Ophthalmologie
July 2024
Zentrum für Augenheilkunde, Universitätsklinikum Köln, Kerpener Str. 62, 50937, Köln, Deutschland.
Due to the demographic changes, the number of older patients in ophthalmological practices and clinics, including those with diplopia, is increasing. Some of the patients report not only horizontally shifted double images but also or only vertically shifted double images. Vertical double vision often causes significant diagnostic problems for ophthalmologists.
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