March 17th 2023
Review top news and interview highlights from the week ending March 17, 2023.
UX111 Shows Continued Dose-Dependent Efficacy as an MPS IIIA Treatment
February 28th 2023Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.