Whether you want more information to aid your management of MPS VI or tools to help your patients, we have many valuable resources to share.
Resources for healthcare professionals
BioMarin is pleased to provide the following clinical reprints, brochures, and practice tools to aid you in managing MPS VI. Simply click on the icon or link to access the desired resource.
- Management guidelines for mucopolysaccharidosis VI.
Giugliani R, Harmatz P, Wraith JE. Pediatrics.2007;120(2):405-418.
- Clinical utility of endurance measures for evaluation of treatment in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
McDonald A, Steiner R, Kuehl K, Turbeville S. J Pediatr Rehabil Med. 2010;3(2):119-127.
- Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
Harmatz P, Yu ZF, Giugliani R, et al. J Inherit Metab Dis. 2010;33(1):51-60.
- Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfastase.
Harmatz P, Giugliani R, Schwartz IV, et al. Mol Genet Metab.2008;94(4):469-475.
- Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy.
Braunlin, Rosenfeld, Kampmann, et al. Journal of Inherited Metabolic Disease. 2013 Mar;36(2):385-94
- Successful management of difficult infusion-associated reactions in a young patient with mucopolysaccharidosis type VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme®]).
Kim KH, Decker C, Burton B. Pediatrics.2008;121(3): e714-e717.
MPS VI clinical surveillance program
BioMarin encourages participation in data collection for the MPS VI Clinical Surveillance Program (CSP). The MPS VI CSP is an ongoing observational database tracking the medical history and outcomes of people with MPS VI.
Advancing MPS VI treatment and research
- Provides a resource to physicians and patients for optimizing patient care
- Evaluates long-term safety and efficacy of NAGLAZYME® (galsulfase)
- Establishes the first comprehensive resource for understanding the variability and progression of MPS VI, as well as the long-term safety and efficacy of the only enzyme replacement therapy indicated for its treatment
- Collects data from multiple centers around the world to compile in an observational database of longitudinal data, available to participating physicians
Optimizing patient care
- Consolidates data to develop new patient-monitoring standards
- Reports patient outcomes
- Provides new understanding for optimizing patient care
- Recommended assessments reflect a high standard of patient care based on feedback from geneticists who have treated MPS VI patients
Easy participation for both physician and patient
- Participation in the CSP is voluntary
- No experimental treatments or assessments
- The only requirements to enroll a patient are a confirmed diagnosis of MPS VI, appropriate institutional approval, and patient or legal guardian consent
To help patients and families manage MPS VI, BioMarin offers several valuable brochures and practical tools. Simply click on the link to access the desired resource.