Artwork

Content provided by Patient Worthy. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Patient Worthy or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.
Player FM - Podcast App
Go offline with the Player FM app!

The Bespoke Gene Therapy Consortium's New Regulatory Playbook, feat. The Foundation for the National Institutes of Health and Taylor's Tale

36:30
 
Share
 

Manage episode 412207562 series 2655724
Content provided by Patient Worthy. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Patient Worthy or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.

In this episode of the podcast we talk 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.

Learn more about the FNIH and the BGTC Regulatory Playbook here.
Learn more about Taylor's Tale and the ways there are helping rare disease patients here.

Editor's Note: Chronic conditions and rare diseases don’t discriminate. Patient Worthy and our partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a story to share, learn more about how your voice can help spread awareness and inspire individuals from all walks of life here: https://shorturl.at/sPV02

  continue reading

65 episodes

Artwork
iconShare
 
Manage episode 412207562 series 2655724
Content provided by Patient Worthy. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Patient Worthy or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.

In this episode of the podcast we talk 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.

Learn more about the FNIH and the BGTC Regulatory Playbook here.
Learn more about Taylor's Tale and the ways there are helping rare disease patients here.

Editor's Note: Chronic conditions and rare diseases don’t discriminate. Patient Worthy and our partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a story to share, learn more about how your voice can help spread awareness and inspire individuals from all walks of life here: https://shorturl.at/sPV02

  continue reading

65 episodes

All episodes

×
 
Loading …

Welcome to Player FM!

Player FM is scanning the web for high-quality podcasts for you to enjoy right now. It's the best podcast app and works on Android, iPhone, and the web. Signup to sync subscriptions across devices.

 

Quick Reference Guide