Ultragenyx Pharmaceutical Inc

Individual Patient Compassionate Use of Mepsevii

Brief Summary: 
Individual patient expanded access requests may be considered for patients who have no other treatment options
Detailed Description: 

Expanded access may provide access for treatment prior to approval by the local regulatory agency. For full details, please visit the link provided.

EA Type
Individual Patients
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome

Drug: Mepsevii
Other Name: UX003
Other Name: recombinant human beta-glucuronidase
Other Name: rhGUS
Other Name: vestronidase alfa

Eligibility Criteria: 

Inclusion Criteria:


Exclusion Criteria:


Eligibility Gender: 

Early Access

Ultragenyx Pharmaceutical Inc
EA Number: 
Expanded Access, Compassionate Use
MeSH Terms: 
Mucopolysaccharidoses, Mucopolysaccharidosis VII
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.