Patient-Focused Drug Development for a Single Intravenous Dose of HMI-203 Gene Therapy in Adult Mucopolysaccharidosis (MPS) II, or Hunter Syndrome, Patients

Patient-Focused Drug Development for a Single Intravenous Dose of HMI-203 Gene Therapy in Adult Mucopolysaccharidosis (MPS) II, or Hunter Syndrome, Patients

Haroldson J, PharmD; Witalisz CJ, MBA; Martin R, MD. et al.