Researchers at the Centre for Genomic Regulation and Wellcome Sanger Institute published evidence that a single, FDA-approved small molecule drug can stabilize nearly all missense variants of the vasopressin V2 receptor (V2R), a G-protein-coupled receptor implicated in rare genetic diseases. The study, appearing in Nature Structural & Molecular Biology, demonstrates that targeted pharmacological chaperones can rescue protein folding and trafficking defects regardless of mutation location. This finding offers a new precision medicine paradigm for treating diverse rare diseases caused by protein misfolding and could accelerate drug repurposing efforts.