Therapeutic advances in neuromuscular diseases in 2023
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Published:2024-01
Issue:1
Volume:23
Page:24-25
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ISSN:1474-4422
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Container-title:The Lancet Neurology
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language:en
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Short-container-title:The Lancet Neurology
Subject
Neurology (clinical)
Reference10 articles.
1. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study;Bril;Lancet Neurol,2023
2. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study;Howard;Lancet Neurol,2023
3. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: safety, tolerability, pharmacokinetics, and target engagement;Mellion;Br J Clin Pharmacol,2021
4. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial;Thornton;Lancet Neurol,2023
5. Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥4 to <8 years) with Duchenne muscular dystrophy: 1-year interim results from study SRP-9001-103 (ENDEAVOR);Zaidman;Ann Neurol,2023