MGNet: Discussing the Impact of Rescue Therapy in Clinical Trials for Myasthenia Gravis

New research from the Myasthenia Gravis Rare Disease Network (MGNet). This summary is based on a paper published in the journal Annals of Clinical and Translational Neurology on April 16, 2025, titled "Handling rescue therapy in myasthenia gravis clinical trials: why it matters and why you should care." 

Read the paper here. 

Learn more about MGNet. 

Transcript: 

New research from the Myasthenia Gravis Rare Disease Network (MGNet), a research group of the Rare Diseases Clinical Research Network.

Discussing the Impact of Rescue Therapy in Clinical Trials for Myasthenia Gravis.

This summary is based on a paper published in the journal Annals of Clinical and Translational Neurology on April 16, 2025.

Myasthenia gravis (MG) is a neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors in muscles, causing disabling weakness. Clinical trials for MG aim to evaluate whether a new therapy can improve patients’ symptoms. However, if a study participant experiences worsening symptoms, they typically receive existing therapies to improve their condition. This type of intervention, called “rescue therapy,” could lead to inaccurate information if not appropriately addressed.

In this review, researchers discuss the impact of rescue therapy in MG clinical trials. The team reviews strategies for incorporating rescue therapy in the design and statistical analysis of MG trials, shows how each strategy affects the interpretation of trial results, and suggests circumstances when each strategy may or may not be applicable to patients or physicians.

Authors note that rescue therapy should be considered during the planning phase of clinical trials before designing the statistical analysis, which can improve the robustness of the study and alignment with regulatory recommendations.