This clinical trial focuses on a rare condition known as Glycogen Storage Disease Type Ia (GSDIa). This is a genetic disorder that affects how the body stores and uses sugar, which can lead to low blood sugar levels and other health issues. The study is examining a treatment called Pariglasgene brecaparvovec, also known by its code name DTX401. This treatment is given as a solution through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein.

The main purpose of the study is to evaluate the long-term safety of DTX401 in patients with GSDIa, as well as to monitor pregnancy outcomes in these patients. Participants in the study may have previously received DTX401 in an earlier clinical trial or as part of their regular treatment. The study will track any serious side effects that might occur, including those related to the infusion process or any other medications the patients might be taking, such as steroids. It will also look at any potential effects on the liver, the development of new or worsening cancers, and other health issues that might arise over time.

Throughout the study, various assessments will be conducted to gather information on the participants’ nutritional status, clinical and laboratory findings, and overall health experiences. This will help researchers understand the impact of DTX401 on patients’ lives and identify any major clinical events that occur during the study period. The study is expected to continue for several years, allowing for comprehensive monitoring and data collection.