This study is about understanding how people feel and what they know about gene therapy for rare diseases. Gene therapy is a new type of treatment that tries to fix or replace broken genes in our bodies. The study will talk to patients, parents, and healthcare workers to find out their thoughts and worries. With this information, an online platform will be created to help families learn more and make better decisions about these treatments.
- Participants will join a one-on-one interview through video calls.
- This study will help create educational resources for families considering gene therapy.
- Interviews will need internet access and a computer or mobile device.