ResearchCon 2020 | Therapeutic Approaches for Rare and Nonsense Mutations

About 10 percent of people with cystic fibrosis–most of whom have two rare and/or nonsense mutations–are not eligible for any of the currently available cystic fibrosis transmembrane conductance regulator (CFTR) protein modulator drugs.

If you are living with a rare or nonsense mutation, if you’re caring for or partnered with someone who is, or if you’re just curious, learn from the experts about the innovative and varied scientific research underway to ensure that 100 percent of people with CF have treatments for the underlying cause of the disease.

Originally recorded at ResearchCon on April 16, 2020.