A goal for all diseases is a treatment that works to prevent, halt, or reverse their effects – essentially, a cure. Achieving this requires early diagnosis, knowledge of disease mechanisms, and effective treatment. For rare diseases, each of these elements is a huge challenge. This perspective explores how real-world and registry data can address these obstacles and considers future developments with the potential for the most significant impact.   Medical Writing. 2025;34(1):46–50. https://doi.org/10.56012/kwle5627