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MPS 7

Clinical trials for MPS 7 explained in plain language.

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  • Global effort launched to map rare genetic disease

    Knowledge-focused Recruiting now

    This study aims to better understand how MPS VII (Sly Syndrome) progresses and how the treatment vestronidase alfa works over the long term. It will follow about 50 patients, both those receiving the treatment and those who are not, to collect detailed health information. The goa…

    Matched conditions: MPS 7

    Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused

    Last updated Mar 16, 2026 15:26 UTC

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