Experimental growth shot tested for kids with rare bone disease

NCT ID NCT05845749

Summary

This early-stage study is testing whether daily injections of a medication called vosoritide can safely help children with rare genetic disorders (MPS IVA and MPS VI) grow taller. Six children aged 5-10 will receive the injections for nearly two years while researchers monitor safety and measure growth changes. Participants must already be receiving standard enzyme replacement therapy and will continue it throughout the study.

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Contacts and locations

Locations

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

Conditions

Explore the condition pages connected to this study.