Date
Thursday, April 16, 2020
Name
Therapeutic Approaches for Rare and Nonsense Mutations
Session Format
Information
Advanced: This session will cover complex topics, so it will be helpful to have a basic knowledge of CF biology.
Description
About 10% of people with cystic fibrosis – most of whom have two rare and/or nonsense mutations – are not eligible for any of the currently available 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, come learn from the experts about the innovative and varied scientific research underway to ensure that 100% of people with CF have treatments for the underlying cause of the disease.