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There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.
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| Measure | Description | Time Frame |
|---|---|---|
| Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03 | This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period. | Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02 |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with MPS VI who previously participated in ASB-00-02
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital and Research Center | Oakland | California | 94609 | United States | ||
| Women's and Children's hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24764221 | Derived | Giugliani R, Lampe C, Guffon N, Ketteridge D, Leao-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24. |
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| ID | Term |
|---|---|
| D009087 | Mucopolysaccharidosis VI |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| North Adelaide |
| Australia |
| Hospital de Clinicas de Porto Alegre | Porto Alegre | 21615 | Brazil |
| Hôpital Femme Mère Enfant | Lyon | Lyon | 69677 | France |
| Prof Michael Beck | Mainz | Mainz | 55131 | Germany |
| Al. Prof Hernani Monteiro | Porto | Porto District | 4202-451 | Portugal |
| Manchester Academic Health Sciences Centre | Manchester | Manchester | M13 9 WL | United Kingdom |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |