Patient Worthy’s award-winning podcast “Wait, How Do You Spell That? A Rare Disease Podcast” is back with a new episode. This week, Colby is sitting down with Drs. Julie Gerberding and Courtney Silverthorn from the Foundation for the National Institutes of Health (FNIH). They’re bringing us updates on the Bespoke Gene Therapy Consortium’s new regulatory playbook that is designed to help get certain types of genetic therapies for rare diseases approved and available to patients more quickly. We’re also joined by Sharon King, the Co-Founder of Taylor’s Tale a non-profit that advocates on behalf of rare disease patients. She talks about why timely development of gene therapies is so important for people like her daughter, who lived with CLN1 disease.
Check out the episode below:
If you want to save this one for later, you can also listen to the episode at this link. Or, you can find the episode on Apple Podcasts, Google Podcasts, Spotify and Amazon. You can also now find our podcast over at the Patient Worthy YouTube channel. Subscribe today!
Editor’s Note: Chronic conditions and rare diseases don’t discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a journey to share, click here to learn more about how your voice can help spread awareness and inspire individuals from all walks of life.
