Clinical trial design for HMI-203 investigational gene therapy for Mucopolysaccharidosis (MPS II) informed by cross-correction potential and KOL Input

Clinical trial design for HMI-203 investigational gene therapy for Mucopolysaccharidosis (MPS II) informed by cross-correction potential and KOL Input

Gingras J, Haroldson J, Smith L, et al. WORLDSymposium 2022.