Comment from Y Liang
AnonymousSupportIndividual
Summary: A relative of a person with a rare genetic mutation supports the proposed framework for developing individualized therapies. The commenter argues that the framework is a promising step for addressing incurable conditions where traditional clinical trials are not feasible.
I am a blood relative of someone with a rare genetic mutation, and I am at risk for inheriting the mutation and also succumbing to this currently incurable condition. The Plausible Mechanism Framework is a promising step toward developing individualized therapies for rare diseases, and I support the ability of all individuals - researchers, doctors, trial participants - to pursue considered paths to therapies where traditional clinical trials are impossible.