Randomized Trialhigh evidence

Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.

Lancet (London, England)·May 2, 2026·PMID 42030965

Why it matters

Cemdisiran offers a novel mechanism—siRNA-mediated C5 inhibition—with convenient quarterly subcutaneous dosing, potentially expanding complement-targeted treatment options beyond current intravenous C5 inhibitors for AChR antibody-positive generalized myasthenia gravis.

Infographic summary of Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.
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