Project Grant R01HL173371

Award Date 7/15/25
Completion Date 4/30/30
Dollars Obligated $852K
Federal Grant Program
93.837
Assistance Type
Project Grant
Place of Performance
California, USA
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  1. Prospectively assess cardiac and arterial structure and function in a large cohort of MPS I and IVA patients compared to healthy controls to establish a MPS cardiovascular disease natural history.

  2. Define and validate prognostic biomarkers of MPS cardiovascular disease in both human and murine models.

  3. Evaluate the efficacy of therapeutic compounds (click-xyloside modulators of GAG biosynthesis and an elastin proteolysis inhibitor) to mitigate cardiovascular manifestations in murine MPS models.

The award period is from Jul 15, 2025 to Apr 30, 2030 and will be performed by the Children's Hospital of Orange County, a prominent non-profit pediatric healthcare institution in California.

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