Over the last decade, highly effective CFTR modulators (HEM) have transformed the health and prospects for a large majority of people with CF. Nevertheless, there remains a substantial need to bring effective CFTR therapies to those with CF who lack treatment options for the underlying cause of their disease as well as those with sup-optimal responses or tolerability issues to approved drugs.
During plenary 3 of NACFC 2021, Manu Jain MD, MSc, will highlight recent data on the real world-impact of HEM as well as data supporting the use of CFTR modulators in new CF communities. He will also discuss emerging data on the pre-clinical and clinical development of genetic therapies that rescue, restore and/or replace CFTR as well as alternative strategies to target the underlying ion transport defects of the disease.
Originally recorded November 5, 2021.