NCT00214773
MPS VI, Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
Any
No
Observational
237
2005-07
2021-04-05
Irvine, California, United States
Los Angeles, California, United States
Oakland, California, United States
Decatur, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Baton Rouge, Louisiana, United States
New Orleans, Louisiana, United States
Baltimore, Maryland, United States
Minneapolis, Minnesota, United States
Minneapolis, Minnesota, United States
Kansas City, Missouri, United States
Paterson, New Jersey, United States
New York, New York, United States
Asheville, North Carolina, United States
Charlotte, North Carolina, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Sioux Falls, South Dakota, United States
Salt Lake City, Utah, United States
Norfolk, Virginia, United States
Seattle, Washington, United States
Wentworthville, New South Wales, Australia
Brisbane, Queensland, Australia
Adelaide, South Australia, Australia
Parkville, Victoria, Australia
Subiaco, Western Australia, Australia
Graz, Steiermark, Austria
Charleroi, Wallonie, Belgium
Bron cedex, Rhône-alpes, France
Paris, , France
Kammin, Mecklenburg-vorpommern, Germany
Hannover, Niedersachsen, Germany
Mainz, Rheinland-Pfalz RP, Germany
Berlin, , Germany
Hamburg, , Germany
Limerick, , Ireland
Cibali, Catania, Italy
Padova, Veneto, Italy
Monza, , Italy
Vilnius, , Lithuania
Rotterdam, , Netherlands
Porto, , Portugal
Stockholm, , Sweden
Birmingham, , United Kingdom
Birmingham, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Salford, , United Kingdom
Inclusion Criteria
All patients must meet the following criteria to qualify for enrollment in the CSP:
* Patient or patient’s parent or legal guardian, if child is under 18 year old or is unable to consent, has provided a signed Patient Information and Authorization Form.
* Patient has laboratory results confirming a diagnosis of MPS VI disease based on detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood spots)and/or abnormality on the ARSB gene.
* Patient is willing to undergo general assessments to establish baseline data or permits physician to enter assessment data recorded prior to CSP entry if available in the patient’s medical records. General assessments include: urinary GAG level, urinary protein level, serum sample for antibody levels, height, weight, and patient history.
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