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Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326

RecruitingN/A

Sponsor: Duke University

Conditions: Pompe Disease

Interventions: Observational

Countries: United States

This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria
Inclusion Criteria:

* Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease
* Must provide a written informed consent
Locations (1)
  • Durham, North Carolina, United States