New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making

BioDrugs, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 27, 2025

Language: Английский

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Real-World Case Series of Efgartigimod for Japanese Generalized Myasthenia Gravis: Well-Tailored Treatment Cycle Intervals Contribute to Sustained Symptom Control

Biomedicines, Journal Year: 2024, Volume and Issue: 12(6), P. 1214 - 1214

Published: May 30, 2024

Introduction: Myasthenia gravis (MG), an immune disorder affecting nerve-muscle transmission, often necessitates tailored therapies to alleviate longitudinal symptom fluctuations. Here, we aimed examine and compare the treatment cycle intervals efficacy of efgartigimod in four patients. This case series mainly offers insights into personalized for patients with MG our facility Japan. Methods: We retrospectively analyzed (2 early-onset, 1 late-onset, seronegative MG, managed oral immunosuppressants as prior treatments) who completed or more cycles from January 2022 September 2023. focused on changes serum immunoglobulin (IgG) level, acetylcholine receptor antibody (AChR-Ab) titer, quantitative (QMG) score. Results: Efgartigimod, administered at a median 5.0 [IQR 5.0, 7.5] weeks between cycles, led decreased IgG levels all reduced AChR-Ab titers seropositive All showed sustained improvement, considerably QMG scores before treatment. None required rescue medications developed treatment-related adverse events. Conclusions: Customized administration effectively enhanced clinical outcomes without notable fluctuations, demonstrating benefits individualized approaches validating safety during study period.

Language: Английский

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Zilucoplan: A Novel Strategy for Management of Myasthenia Gravis

American Journal of Therapeutics, Journal Year: 2025, Volume and Issue: 32(2), P. e138 - e142

Published: Feb. 27, 2025

Background: Generalized myasthenia gravis is an autoimmune disease that leads to eventual end-organ damage. Zilucoplan a new agent for the treatment of generalized patients who are acetylcholine receptor antibody positive. Pharmacodynamics, And Pharmacokinetics: It first in its class exerts effect by binding complement protein C5 and inhibiting activation C5a C5b-9. may increase risk bacterial infections patients, as mechanism does heavily rely on altering components immune system. has black box warning because meningococcal infections. subcutaneous, at-home gravis, which allow increased adherence. supplied prefilled syringe dosing varies based bodyweight. Onset action was seen early 1 week clinical studies. Clinical Trials: There were 2 major trials led Food Drug Administration approval. A 12-week phase study identified 0.3 mg/kg clinically efficacious dose with gravis. 3 trial (RAISE) showed benefit randomized zilucoplan compared placebo. significant change Myasthenia Gravis Activities Daily Living score after 12 weeks treatment. no difference safety end points both Most experienced treatment-emergent adverse events, COVID-19 infections, exacerbation symptoms being among most commonly reported. Therapeutic Advance: novel provides additional option do not respond standard care. Evidence from postmarketing studies assessing benefit–risk profile real-world settings, addition controled trials, well pharmacoeconomic studies, necessary establish place therapy.

Language: Английский

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What Is in the Neuromuscular Junction Literature?

Journal of Clinical Neuromuscular Disease, Journal Year: 2024, Volume and Issue: 26(2), P. 90 - 99

Published: Nov. 18, 2024

Abstract This update starts with an interesting series of children and adults congenital myasthenic syndrome a DOK7 variant. The next section is on autoimmune myasthenia gravis (MG) epidemiology, cost care, hospitalizations. A number studies the newer treatments are discussed including phase 2 trial nipocalimab recommendations for using some these drugs. large emphasizing negative effects pyridostigmine in muscle-specific kinase MG covered. study incidence taste disorders alopecia follows. ends topic burden disease Lambert–Eaton syndrome.

Language: Английский

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Usability of the zilucoplan prefilled syringe for the treatment of generalized myasthenia gravis: insights from a human factors validation study

Expert Review of Medical Devices, Journal Year: 2024, Volume and Issue: 21(10), P. 977 - 982

Published: Oct. 2, 2024

Generalized myasthenia gravis (gMG) is a chronic, autoimmune neuromuscular disease, often accompanied by high treatment burden. The objective of this usability study was to validate that the zilucoplan prefilled syringe (PFS) can be used safely and effectively.

Language: Английский

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