#29. Cystic Fibrosis: A Blueprint for Rare Disease Cures
Description
In this podcast episode, we explore how cystic fibrosis (CF) evolved from a fatal childhood illness to a manageable chronic condition, thanks to groundbreaking therapies targeting its molecular roots. Highlighting the development of CFTR modulator drugs like Trikafta, we discuss decades of multidisciplinary collaboration, innovative funding models, and cutting-edge technologies that made this possible. The episode also celebrates the 2025 Lasker~DeBakey Clinical Medical Research Award, honoring Michael J. Welsh, Jesús González, and Paul A. Negulescu for their pivotal roles in discovering and developing these transformative treatments. Finally, we reflect on how these achievements serve as a blueprint for advancing cures for other rare diseases, with AI poised to play a key role in the next era of discovery. Produced by Dr. Jake Chen.
United States
