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This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MPS IVA | Subjects with mucopolysaccharidosis IVA (Morquio Syndrome) |
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| Measure | Description | Time Frame |
|---|---|---|
| Endurance | Study Visit | |
| Respiratory Function | Study Visit |
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Inclusion Criteria:
Exclusion Criteria:
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Subjects diagnosed with MPS IVA
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| Name | Affiliation | Role |
|---|---|---|
| Celeste Decker, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oakland | California | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21877895 | Derived | Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172. |
| Label | URL |
|---|---|
| BioMarin Pharmaceutical Inc. website | View source |
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| Chicago |
| Illinois |
| United States |
| Córdoba | Argentina |
| Porto Alegre | Brazil |
| São Paulo | Brazil |
| Montreal | Quebec | Canada |
| Lyon | France |
| Paris | France |
| Mainz | Germany |
| Monza | Italy |
| Amsterdam | Netherlands |
| Taipei | Taiwan |
| Birmingham | United Kingdom |
| London | United Kingdom |
| Manchester | United Kingdom |
| ID | Term |
|---|---|
| D009085 | Mucopolysaccharidosis IV |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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