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Expert Interview

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

A Second View: Examining the achondroplasia treatment landscape with a focus on BBIO's infigratinib, BMRN's Voxzogo (vosoritide) & ASND's TransCon CNP

Ticker(s): BBIO, BMRN, ASND

Who's the expert?

Institution: University Hospital Cologne

  • Head of the Center for Rare Skeletal Disorders in Children and Adolescents at the University of Cologne & member of the Osteogenesis Imperfecta Federation Europe (OIFE) Medical Advisory Board
  • Treats 52 patients with Achondroplasia older than 5 years of age and is currently prescribing Voxzogo to 26 patients.
  • Specializes in the research of brittle bone disease and directs the special ambulance for rare pediatric skeletal diseases and pediatric rheumatology.

Interview Questions

Please describe your clinical practice, in particular with treating achondroplasia patients, and research in the space.

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How many patients do you treat with achondroplasia? What treatment options do they have?

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What is the mechanism of action of a C-type natriuretic peptide like vosoritide, how does it regulate bone growth?

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Change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr. How satisfactory is this result?

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