In this episode of Rare on Air, host Julien Poulain delves back into the world of gene therapies, chatting with Khadidja Hadri, a French mother whose nine-year-old son grapples with the rare genetic disorder ADA-SCID, and Stefano Benvenuti, Public Affairs Manager at the Fondazione Telethon in Italy.
Khadidja candidly shares how a groundbreaking gene therapy transformed her son’s health, significantly altering the trajectory of their lives.
Stefano provides insights into the workings of gene therapies, detailing the one administered to Khadidja's son, and also discusses his perspectives on proposed EU reforms to boost incentives for rare disease medicine development.
Connect with the podcast at [email protected]⁠, and join the dialogue on social media using #EurordisRareOnAir!
Also, don't forget to secure your spot at the 12th edition of the European Conference on Rare Diseases and Orphan Products (ECRD 2024), taking place online and in Brussels on 15-16 May 2024. Register for the event at: https://www.rare-diseases.eu/.

Ver todos los episodios