Expanded Access to Mepsevii
- Conditions
- MPS VIIMucopolysaccharidosis VIISly Syndrome
- Registration Number
- NCT03775174
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- Brief Summary
Individual patient expanded access requests may be considered for patients who have no other treatment options
- Detailed Description
Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below.
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
Exclusion Criteria
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method