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Rozanolixizumab for Myasthenia Gravis: a breakthrough treatment and future prospects.

作者信息

Matic Alexandria, Bril Vera

机构信息

The Ellen & Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.

出版信息

Immunotherapy. 2025 Apr;17(5):309-316. doi: 10.1080/1750743X.2025.2491295. Epub 2025 Apr 25.


DOI:10.1080/1750743X.2025.2491295
PMID:40277145
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12045563/
Abstract

Myasthenia gravis is a rare chronic autoimmune disorder affecting the post-synaptic neuromuscular junction, primarily mediated by pathogenic immunoglobulin G (IgG) targeting specific proteins like acetylcholine receptor (AChR), muscle-specific tyrosine kinase (MuSK), and low-density lipoprotein receptor-related protein 4 (LRP4). Modulating pathogenic IgG is a promising approach for disease management. Rozanolixizumab, a human IgG4 neonatal Fc receptor (FcRn) inhibitor, enhances the degradation of pathogenic IgG by 78%, marking a significant advancement in treating generalized myasthenia gravis. It offers effective management for patients with AChR or MuSK antibodies and is administered subcutaneously with mild to moderate adverse events. However, the safety and efficacy of rozanolixizumab require further validation through real-world post-marketing studies. If current trial results are confirmed, rozanolixizumab may become a preferred treatment option for myasthenia gravis in the near future. This review examines clinical trials evaluating the pharmacokinetics, efficacy, and safety of rozanolixizumab in patients with generalized myasthenia gravis and discusses ongoing trials and future research directions.

摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b98d/12045563/f56946826fa7/IIMY_A_2491295_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b98d/12045563/f56946826fa7/IIMY_A_2491295_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b98d/12045563/f56946826fa7/IIMY_A_2491295_F0001_OC.jpg

相似文献

[1]
Rozanolixizumab for Myasthenia Gravis: a breakthrough treatment and future prospects.

Immunotherapy. 2025-4

[2]
Rozanolixizumab in generalized myasthenia gravis: Pooled analysis of the Phase 3 MycarinG study and two open-label extensions.

J Neuromuscul Dis. 2025-3

[3]
Efficacy 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. 2024-9-12

[4]
An evaluation of rozanolixizumab-noli for the treatment of anti-AChR and anti-MuSK antibody-positive generalized myasthenia gravis.

Expert Opin Biol Ther. 2023

[5]
Long-term safety of cyclical rozanolixizumab in patients with generalized myasthenia gravis: Results from the Phase 3 MycarinG study and an open-label extension.

J Neuromuscul Dis. 2025-3

[6]
Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration.

MAbs. 2018-9-12

[7]
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.

Lancet Neurol. 2023-5

[8]
The efficacy and safety of FcRn inhibitors in patients with myasthenia gravis: a systematic review and meta-analysis.

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[9]
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[10]
MuSK myasthenia gravis IgG4 disrupts the interaction of LRP4 with MuSK but both IgG4 and IgG1-3 can disperse preformed agrin-independent AChR clusters.

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本文引用的文献

[1]
Long-term safety of cyclical rozanolixizumab in patients with generalized myasthenia gravis: Results from the Phase 3 MycarinG study and an open-label extension.

J Neuromuscul Dis. 2025-3

[2]
Initiation response, maximized therapeutic efficacy, and post-treatment effects of biological targeted therapies in myasthenia gravis: a systematic review and network meta-analysis.

Front Neurol. 2024-10-28

[3]
Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis.

Adv Ther. 2024-12

[4]
Efficacy 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. 2024-9-12

[5]
Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion.

Neurol Sci. 2024-12

[6]
B cell-targeting chimeric antigen receptor T cells as an emerging therapy in neuroimmunological diseases.

Lancet Neurol. 2024-6

[7]
Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study.

J Neurol Neurosurg Psychiatry. 2024-8-16

[8]
FcRn Inhibitor Therapies in Neurologic Diseases.

CNS Drugs. 2024-6

[9]
The efficacy and safety of FcRn inhibitors in patients with myasthenia gravis: a systematic review and meta-analysis.

J Neurol. 2024-5

[10]
An evaluation of rozanolixizumab-noli for the treatment of anti-AChR and anti-MuSK antibody-positive generalized myasthenia gravis.

Expert Opin Biol Ther. 2023

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