From the Rare Disease Bunker to Many More Birthdays – A Tale of a Gene Therapy that Cures her Daughter with AADC Deficiency – The First Spanish Patient – with Carolina Moreno
ONCE UPON A GENE – EPISODE 192
From the Rare Disease Bunker to Many More Birthdays – A Tale of a Gene Therapy that Cures her Daughter with AADC Deficiency – The First Spanish Patient – with Carolina Moreno
Enjoy this presentation by rare disease mom, Carolina Moreno, which she originally shared at the 2023 International CTNNB1 conference held in Spain.
LINKS & RESOURCES MENTIONED
Ahora sí, te quiero tal como eres / Now yes, I love you as you were
https://neret.cat/producte/ahora-si-te-quiero-tal-como-eres/
https://fundacioncolumbus.org/
First patient treated for AADC deficiency with gene therapy by Columbus Foundation