Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

• Main Authors: Carsten W. Lederer (Author), Lola Koniali (Author), Tina Buerki-Thurnherr (Author), Panayiota L. Papasavva (Author), Stefania La Grutta (Author), Amelia Licari (Author), Frantisek Staud (Author), Donato Bonifazi (Author), Marina Kleanthous (Author)
• Format: Book
• Published: MDPI AG, 2022-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 disorders with pre- or perinatal onset, timely interventi...