About Maroteaux-Lamy Syndrome
Maroteaux-Lamy syndrome (MPS VI) is caused by deficiency of arylsulfatase B (ARSB), resulting in accumulation of dermatan sulfate in lysosomes throughout the body. Unlike MPS I (Hurler), intelligence is generally preserved. The disease causes severe skeletal dysplasia, cardiac valve disease, corneal clouding, obstructive airway disease, and hepatosplenomegaly. Galsulfase (Naglazyme), the approved enzyme replacement therapy, significantly improves endurance and respiratory function.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Maroteaux-Lamy Syndrome trials.
Galsulfase (Naglazyme) ERT is standard — document infusion history, antibody status, and any infusion reactions
Urinary dermatan sulfate and ARSB enzyme activity are required biomarkers for eligibility documentation
Six-minute walk test and pulmonary function tests are standard outcome measures — baseline values are eligibility determinants
Cardiac valve assessment (echocardiogram) is required — severe cardiac disease may affect anesthesia eligibility for trial procedures
Patient Resources
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