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This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Myozyme | Biological | 10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow |
|
| Measure | Description | Time Frame |
|---|---|---|
| The objective of this extension study was to monitor the long-term safety and efficacy | 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 |
|---|---|---|---|---|---|---|
| Children's Hospital of Oakland | Oakland | California | United States | |||
| Children's Hospital of Orange Country |
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| Orange |
| California |
| United States |
| University of Nebraska Medical Center | Omaha | Nebraska | United States |
| Institute for Genetic Medicine, Saint Peter's University Hospital | New Brunswick | New Jersey | United States |
| New York University [NYU] School of Medicine | New York | New York | United States |
| Pediatrique Hospital Debrousse | Lyon | France |
| Hôpital Porte Madeleine | Orléans | France |
| The Morningside Clinic | Johannesburg | South Africa |
| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C509951 | GAA protein, human |
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