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
Data on HMI-203 from animal studies support its further development for treating MPS II Feb. 12, 2021
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