Mucopolysaccharidosis VII Disease Monitoring Program

The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global,
prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease
presentation and progression, assess long-term effectiveness and safety of vestronidase alfa,
including hypersensitivity reactions and immunogenicity , as well as prospectively
investigate longitudinal change across biomarker(s), clinical assessments, and patient/
caregiver-reported outcome measures in a representative population. The aim of this DMP is to
collect data on patients with MPS VII to provide a comprehensive dataset on the clinical
presentation, heterogeneity, and disease progression, and meaningful standardized ICH
GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a
randomized study and both treated and untreated patients will be enrolled.

Inclusion Criteria:

- Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or
mutation analysis.

- Willing and able to provide written informed consent or, in the case of patients under
the age of 18 (or 16 years, depending on the region) or patients >18 years of age who
have cognitive deficiencies, provide written assent (if required) and written informed
consent by a legally authorized representative after the nature of the DMP has been
explained, and prior to any research-related procedures.

- Willing to comply with DMP visit schedule.

Exclusion Criteria:

- Concurrent enrollment in an Ultragenyx-sponsored clinical trial.

- Participation in any other pharmaceutical company-sponsored interventional clinical
trial unless approved by Ultragenyx.