The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.
Age
2–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible 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 be 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
Clareo Health | Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
