… Current treatments include hematopoietic stem cell transplantation and
enzyme replacement therapy, but these do not address all manifestations of the disease. We …
Prevention of Cardiac, Neurologic, and Skeletal Manifestations of Murine MPS I by Either Systemic or CSF-Directed AAV9-IDUA/RGX-111 Administered at Minimal …
Published: 11/06/2024