Mucopolysaccharidosis VII Disease Monitoring Program

This Disease Monitoring Program (DMP) collects long-term natural history and treatment outcome data on individuals with Mucopolysaccharidosis VII (MPS VII, also known as Sly disease) — an extremely rare inherited metabolic disorder. MPS VII is caused by a deficiency of the enzyme beta-glucuronidase, which leads to the buildup of complex sugars in cells and progressive damage to multiple organs. MPS VII affects the skeleton, organs, vision, hearing, and intellectual development, and very few people worldwide have this condition. The DMP gathers real-world data to better understand disease progression, how well patients respond to treatments, and what measures best capture changes in health status. Any individual with a confirmed MPS VII diagnosis by enzyme or genetic testing, of any age, is eligible. No experimental treatment is involved. You may be eligible if you: - Have been diagnosed with Mucopolysaccharidosis VII (MPS VII / Sly disease) - Have had your diagnosis confirmed by enzyme activity testing or genetic mutation analysis - Are willing to attend regular monitoring visits - Are willing to provide informed consent (or parental/guardian consent if under 18) You may NOT be eligible if you: - Are currently enrolled in another pharmaceutical company-sponsored interventional clinical trial (unless approved by Ultragenyx) Talk to your doctor to see if this trial is right for you.