All articles tagged with #angelman syndrome
Ultragenyx's Phase III Aspire trial of apazunersen in Angelman syndrome missed both its primary endpoint and a key secondary endpoint, prompting a ~43% after-hours stock drop; the company will review the program and pursue significant expense reductions, while analysts warn commercialization could be challenging even if other Angelman studies meet endpoints.
Ultragenyx said its experimental Angelman syndrome therapy GTX-102 showed no benefit versus a sham in a large Phase 3 trial, ending earlier hopes from earlier results and creating a major hurdle for the company's profitability plans in the rare-disease space.
Annie Morgan’s days caring for Ava, who has Angelman syndrome, reveal the mounting emotional and physical toll of long-term caregiving, amplified by a patchwork of support as the U.S. shifts toward family-centered care.
Yale researchers secured a $40M NIH grant to advance STEP, a 10-nm brain-delivery platform for CRISPR gene editors; early preclinical work in mice hints at brain-wide delivery and functional gains, but the mechanism remains unclear, safety and scaling concerns persist, and full details won’t be published until 2028 as clinical work progresses.