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This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Myozyme | Biological | 30 mg/kg qow f and 40 mg/kg qow |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| The objective of this extension study was to monitor the long-term safety and efficacy of a single patient | 3 years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Texas Health Science Center at San Antonio | San Antonio | Texas | United States |
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| ID | Term |
|---|---|
| C509951 | GAA protein, human |
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