March 17th 2023
Review top news and interview highlights from the week ending March 17, 2023.
March 15th 2023
March 10th 2023
Gene Therapy Shows Promise in Infantile GM1 GangliosidosisMarch 8th 2023
Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.
UX111 Shows Continued Dose-Dependent Efficacy as an MPS IIIA TreatmentFebruary 28th 2023
Ultragenyx’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.