SCIE.14.1

15:30

Duchenne Muscular Dystrophy: from natural history to innovative experimental therapies

Mercuri E.M. *

SCIE.14.2

16:00

SR-TIGET Clinical trial updates

Aiuti A. *

SCIE.14.3

16:30

Updates of two AAV-mediated, liver-directed gene therapy clinical trials

Brunetti Pierri N. *