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Expanded Access to Mepsevii

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ClinicalTrials.gov Identifier: NCT03775174
Expanded Access Status : Available
First Posted : December 13, 2018
Last Update Posted : November 23, 2022
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

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

Condition or disease Intervention/treatment
MPS VII Mucopolysaccharidosis VII Sly Syndrome Drug: Mepsevii

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.

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.

Intervention Details:
  • Drug: Mepsevii
    Other Names:
    • UX003
    • recombinant human beta-glucuronidase
    • rhGUS
    • vestronidase alfa

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

Inclusion Criteria:


Exclusion Criteria:


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03775174

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Contact: Early Access 1-415-483-8800 EarlyAccess@ultragenyx.com

Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Additional Information:
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03775174    
Other Study ID Numbers: UX003-EAP
First Posted: December 13, 2018    Key Record Dates
Last Update Posted: November 23, 2022
Last Verified: November 2022
Keywords provided by Ultragenyx Pharmaceutical Inc:
Expanded Access
Compassionate Use
Additional relevant MeSH terms:
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Mucopolysaccharidosis VII
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases