05:02 PM EDT, 09/08/2025 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) reported positive longer-term results from its Phase 3 study of a gene therapy developed to treat glycogen storage disease type Ia, a rare and life-threatening metabolic disorder.

The investigational therapy DTX401 led to further reductions in patients' reliance on cornstarch supplements at week 96, while maintaining stable blood sugar levels and improving fasting tolerance, the company said Monday in a statement.

"These results are consistent with the positive data observed in the Phase 1/2 study and underscore the robustness and progressive improvement of treatment effect that can potentially be achieved with this gene therapy," Chief Medical Officer Eric Crombez said in the statement.

The gene therapy was well tolerated with an acceptable safety profile, the company said.