The triple-drug combination Trikafta, developed by Paul Negulescu, Sabine Hadida, and Fredrick Van Goor, has been awarded the prestigious Breakthrough prize for its life-changing impact on people with cystic fibrosis. Trikafta has significantly increased life expectancy for those with the inherited disorder and has been hailed as a remarkable achievement in biomedical research. The drugs in Trikafta work together to help a faulty protein function correctly, addressing the root cause of the disease. In addition to this breakthrough, the Breakthrough prize also recognized the discovery of genetic risk factors for Parkinson’s disease and advancements in CAR-T-cell immunotherapy for leukemia. The winners in physics and mathematics were also honored.
https://www.nature.com/articles/d41586-023-02890-1