1. Clinical trial design in gene therapy for neurodegenerative diseases: Sanfilippo A syndrome. Issue 5 (2020) Authors: Morte, Adelaida; Ortiz, Esther; Sust, Mariano; Vaque, Anna; Gascon, Neus; Plata-Salaman, Carlos Journal: Cell & gene therapy insights Issue: Volume 6:Issue 5(2020) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗