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Expert Interview

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

Digging into the recent Phase 2 Cohort 5 results of Bridgebio's ifigratinib in achondroplasia

Ticker(s): BBIO, BMRN, ASND

Who's the expert?

Institution: Hôpital Femme-Mère-Enfant

  • Professor of Pediatrics and Head of the Division of Pediatric Endocrinology at Hôpital Femme-Mère-Enfant (Lyon, France)
  • Treats ~12 patients with achondroplasia.
  • Research focuses on a variety of endocrinological conditions in children and was a co-author on the PROPEL studies of Infigratinib in Children with Achondroplasia.

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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