Valbiotis gains French clearance to begin phase II study of TOTUM-070 to reduce LDL-cholesterol Feb. 16, 2021
Interim phase I/II data show safety and systemic efficacy of RGX-121 in patients with MPS II Feb. 16, 2021
Promising preclinical data for SIG-005 encapsulated nonviral cell-based therapy for MPS Feb. 15, 2021
New phase I/II study evaluates hLB-001 in pediatric patients with severe methylmalonic acidemia Feb. 15, 2021