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Clinical Trial Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.


Clinical Trial Description

Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270). ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05768386
Study type Observational
Source BioMarin Pharmaceutical
Contact
Status Enrolling by invitation
Phase
Start date January 1, 2023
Completion date January 2040

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