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The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Observational | No intervention. This is an observational program. |
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| Measure | Description | Time Frame |
|---|---|---|
| To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase. | at least 15 years |
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Inclusion Criteria
All patients must meet the following criteria to qualify for enrollment in the CSP:
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All patients with a confirmed diagnosis of MPS VI disease may participate in the CSP. It is not a requirement that the patients enrolled in the CSP receive Galsulfase 1mg/kg to participate as this is an observational program.
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| Name | Affiliation | Role |
|---|---|---|
| Julie Johnson | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Irvine | Irvine | California | 92697 | United States | ||
| Children's Hospital of Los Angeles |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27339555 | Derived | Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR; CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab. 2016 Aug;118(4):310-8. doi: 10.1016/j.ymgme.2016.06.001. Epub 2016 Jun 3. |
| Label | URL |
|---|---|
| BioMarin Pharmaceutical Inc Website | View source |
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Urine and serum
| Los Angeles |
| California |
| 90027 |
| United States |
| Children's Hospital and Research Center Oakland | Oakland | California | 94609 | United States |
| Emory University | Decatur | Georgia | 30033 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Indiana University School of Medicine | Indianapolis | Indiana | 46202 | United States |
| Baton Rouge Clinic | Baton Rouge | Louisiana | 70808 | United States |
| Tulane University Medical Center | New Orleans | Louisiana | 70112 | United States |
| Johns Hopkins Univeristy School of Medicine | Baltimore | Maryland | 21287 | United States |
| Children's Health Care | Minneapolis | Minnesota | 55404 | United States |
| University of Minnesota - Fairview University Medical Center | Minneapolis | Minnesota | 55455 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| St. Joseph's Healthcare | Paterson | New Jersey | 07503 | United States |
| New York University | New York | New York | 10016 | United States |
| Fullerton Genetic Center | Asheville | North Carolina | 28803 | United States |
| Carolinas Medical Center | Charlotte | North Carolina | 28203 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Sioux Valey Children's Speciality Clinics | Sioux Falls | South Dakota | 57117 | United States |
| University of Utah Medical Center | Salt Lake City | Utah | 84132 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Westmead Hospital | Wentworthville | New South Wales | NSW2145 | Australia |
| Royal Children's Hospital, Brisbane | Brisbane | Queensland | 4029 | Australia |
| Women and Children's Hospital | Adelaide | South Australia | 5006 | Australia |
| Royal Children's Hospital | Parkville | Victoria | Australia |
| Princess Margaret Hospital for Children | Subiaco | Western Australia | 6008 | Australia |
| LKH-Universitätsklinik Graz, Kinderklinik | Graz | Styria | A-8036 | Austria |
| Institute of Pathology and Genetics, Metabolic Unit | Charleroi | Wallonia | 6041 | Belgium |
| Hôpital Femme Mère Enfant | Bron | Auvergne-Rhône-Alpes | 69677 | France |
| Hopital Necker - Enfants Malades | Paris | France |
| Oberarzt Pädiatrie | Hanover | Lower Saxony | 30173 | Germany |
| Universitatsklinikum Freiburg, Klinik II | Kammin | Mecklenburg-Vorpommern | 79106 | Germany |
| Universitätsmedizin | Mainz | Rheinland-Pfalz RP | 55131 | Germany |
| Charité-Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Universitätsklinikum Hamburg Eppendorf | Hamburg | 20246 | Germany |
| MidWestern Regional Hospital | Limerick | Ireland |
| Azienda Ospedaliero Universitaria | Cibali | Catania | 95123 | Italy |
| Department of Woman's and Child's Health | Padova | Veneto | 35128 | Italy |
| Università Milano Bicocca, Resp. Centro "Fondazione Mariani" | Monza | 20052 | Italy |
| Vilnius University Hospital, Santariskiu Klinikos | Vilnius | LT-08661 | Lithuania |
| Rotterdam University Hospital - Sophia's Children's Hospital | Rotterdam | 3015 GE | Netherlands |
| Hospital de Sao Joao, Unidae de Doencas Metabolicas | Porto | 4200-319 | Portugal |
| Astrid Lindgrens Children's Hospital | Stockholm | Sweden |
| Queen Elizabeth Hospital, University Hospitals Birmingham NHS | Birmingham | B29 6JD | United Kingdom |
| Birmingham Children's Hospital, Steelhouse | Birmingham | B4 6NH | United Kingdom |
| Great Ormond Street Hospital For Children, NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| St Mary's Hospital, Willink Biochemical Genetics Unit | Manchester | M13 9WL | United Kingdom |
| Salford Royal Hospital NHS Trust | Salford | M6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D009087 | Mucopolysaccharidosis VI |
| 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 |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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