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.
Available
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
All
Contacts
Early Access
1-415-483-8800
EarlyAccess@ultragenyx.com
Ultragenyx Pharmaceutical Inc
NCT Number
Keywords
Expanded Access
Compassionate Use
MeSH Terms
Mucopolysaccharidoses
Mucopolysaccharidosis VII