Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial

RCT (n=44 in US) found that zilucoplan, a subcutaneously self-administered macrocyclic peptide inhibitor of complement component 5, led to sustained improvements over 12 weeks, meeting the prespecified primary efficacy end point and demonstrated a favourable safety profile.


JAMA Neurology