In gene therapy, Ultragenyx anticipates data disclosure for its Wilson disease program and plans to file for approval in Glycogen Storage Disease Type Ia (GSD1a) in 2025. The rare disease market ...

In gene therapy, Ultragenyx anticipates data disclosure for its Wilson disease program and plans to file for approval in Glycogen Storage Disease Type Ia (GSD1a) in 2025. The rare disease market ...

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025 Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for ...

As can be seen in the chart above, the company also has ... a Phase 3 study around its candidate DTX401 to treat Glycogen storage disease type I or GSD1a. This is a rare, inherited disorder ...

Here, we found that inhibition of AMPK occurs during naïve transition with two conventional inhibitors of the MEK1 and GSK3β pathways. The accumulation of glycogen due to iGSK3β is responsible for ...

The best cloud storage allows you to free up space on your smartphone, laptop and other devices as well as store an extra copy of your most important files in the cloud. While a free tier is a ...

Besides BEAM-101, BEAM is also expanding its genetic disease pipeline by developing BEAM-301 and BEAM-302 for treating glycogen storage disease type 1a (GSD1a) and alpha-1 antitrypsin deficiency ...

The Official UK Singles Chart reflects the UK’s biggest songs of the week, based on audio and video streams, downloads, CDs and vinyl, compiled by the Official Charts Company. The UK Top 40 is ...