Navigating Rare Disease Trials: Patient Tips for Accessing Novel Therapies
With a 22% rise in rare disease trials in 2024, patients face complex paths to novel therapies. This analysis explores adaptive trial designs, genetic testing insights, and patient-driven strategies shaping access to ultra-rare disorder treatments today and tomorrow.
Robert Maxwell
Aug 07, 2025
orphan disease therapy
rare genetic conditions
ultra-rare disorder studies
patient-centered research
