How CFTR Modulators Work for People with One F508del Mutation

Learn about the science behind the cystic fibrosis drugs that are in development for people with just one copy of the F508del mutation.

[@arnoldronning5471]

I graduated from pediatric residency in 1999. Our CF patients from that time and their suffering re still etched in my memory. Even then, genetics was offering hope for future breakthroughs. Though technically not gene therapy, this molecular level therapy is such a breakthrough. It is an answer to decades of hard work and family prayers. Thank you for sharing.

[@RK-ep8qy]

great video

[@sampathbolla2790]

Great one ❤

[@MikT81]

❤