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Expert Interview

Slingshot members are talking to an expert! The topic is:

A look at Ultragenyx setrusumab (UX143) for the treatment of osteogenesis imperfecta ahead of the Phase 3 clinical trial readout

Ticker(s): RARE, MREO

Who's the expert?

Institution: Children’s Mercy Kansas City

  • Clinical geneticist specializing in rare bone disorders including hypophosphatasia and osteogenesis imperfecta, with board certifications in pediatrics, internal medicine, and clinical genetics
  • Treats a broad population of patients with skeletal dysplasias and other complex genetic bone diseases each year through the Skeletal Dysplasia Clinic
  • Primary research focus is on the diagnosis and treatment of genetic bone disorders, with extensive contributions to the field including more than 70 publications

Interview Questions
Q1.

How would you compare the mechanism of action of setrusumab, as an anti-sclerostin antibody, to current standards of care such as bisphosphonates or teriparatide in pediatric and young adult OI patients?

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Q2.

What data from the Phase 3 portion of the ORBIT study would be most important for you when considering setrusumab for routine clinical use?

Added By: sara_admin
Q3.

What unmet needs remain in the management of OI across different subtypes and age groups, and how do you see a therapy like setrusumab potentially changing long-term care?

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Reason

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