RecruitingGlycogen Storage Disease Type Ia

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Eligible age

2+ yrs

Accepts

All genders

Locations

9 states

Healthy volunteers

See if you qualify for this study

Answer a few quick questions about your location and health. Takes about a minute.

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About this study

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

Sponsor: Ultragenyx Pharmaceutical Inc

You may qualify if…

✓ Patient who had:
✓ DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
✓ Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
✓ Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.

You may not qualify if…

✕ Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator