InternationalResearch and clinical trials
Cemdisiran, given every three months, reduced myasthenia gravis symptoms in a phase 3 trial
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
The NIMBLE study, published in The Lancet, examined cemdisiran in myasthenia gravis. The study was carried out at 86 centers in 13 countries. The drug was injected under the skin every 12 weeks. At 24 weeks, the daily living score improved by 4.5 points with the drug and by 2.2 points with placebo. No serious infections were seen in the cemdisiran group.
What does it mean for patients and families?
This drug is not yet approved. A new option given by infrequent injections may become available in the future.
Source
Scientific paper: Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial. The Lancet, 2026. DOI: 10.1016/S0140-6736(26)00690-2 · PubMed 42030965
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Myasthenia gravis (MG)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.