- Review
Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies
- Carsten W. Lederer,
- Lola Koniali,
- Tina Buerki-Thurnherr,
- Panayiota L. Papasavva,
- Stefania La Grutta,
- Amelia Licari,
- Frantisek Staud,
- Donato Bonifazi and
- Marina Kleanthous
Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorde...