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(Rødgaard A, Nielsen FC, Djurup R, Somnier F, Gammeltoft S. Acetylcholine receptor antibody in myasthenia gravis: predominance of IgG subclasses 1 and 3. Clin Exp Immunol. 1987;67(1):82-88. https://www.ncbi.nlm.nih.gov/pubmed/3621677.3621677)
Rødgaard A, Nielsen FC, Djurup R, Somnier F, Gammeltoft S. Acetylcholine receptor antibody in myasthenia gravis: predominance of IgG subclasses 1 and 3. Clin Exp Immunol. 1987;67(1):82-88. https://www.ncbi.nlm.nih.gov/pubmed/3621677.3621677Rødgaard A, Nielsen FC, Djurup R, Somnier F, Gammeltoft S. Acetylcholine receptor antibody in myasthenia gravis: predominance of IgG subclasses 1 and 3. Clin Exp Immunol. 1987;67(1):82-88. https://www.ncbi.nlm.nih.gov/pubmed/3621677.3621677, Rødgaard A, Nielsen FC, Djurup R, Somnier F, Gammeltoft S. Acetylcholine receptor antibody in myasthenia gravis: predominance of IgG subclasses 1 and 3. Clin Exp Immunol. 1987;67(1):82-88. https://www.ncbi.nlm.nih.gov/pubmed/3621677.3621677
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Key PointsQuestionWhat are the clinical effects of zilucoplan, a subcutaneously self-administered macrocyclic peptide inhibitor of complement component 5, in a broad population of patients with moderate to severe acetylcholine receptor autoantibody–positive generalized myasthenia gravis? FindingsIn a randomized, double-blind, placebo-controlled, multicenter phase 2 trial, zilucoplan yielded rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary end points. Near-complete complement inhibition was associated with a faster onset and greater magnitude of benefit than submaximal complement inhibition, and favorable safety and tolerability were observed. MeaningThe findings support a potential therapeutic role for zilucoplan in generalized myasthenia gravis and further evaluation in a phase 3 study.
JAMA Neurology – American Medical Association
Published: May 17, 2020
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