Pompe Disease Registry
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- STATUS
- Recruiting
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- End date
- Jan 31, 2034
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- participants needed
- 2000
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- sponsor
- Genzyme, a Sanofi Company
Summary
- To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
- To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
- To characterize and describe the Pompe disease population as a whole; and
- To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme.
Description
Details
| Condition | pompe disease |
|---|---|
| Age | 100 years and younger |
| Clinical Study Identifier | NCT00231400 |
| Sponsor | Genzyme, a Sanofi Company |
| Last Modified on | 19 February 2024 |
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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