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Expert Interview

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

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: Kentucky Children's Hospital 

  • Clinical geneticist and Chief, Division of Pediatric Genetics and Metabolism at Kentucky Children's Hospital; formerly Associate Professor of Pediatric Genetics at University of Arkansas for Medical Sciences.
  • Manages 5 patients with achondroplasia in additton to patients with AKT pathway issues.
  • Research led to a report with the first cohort of patients with SATB2-associated syndrome (SAS) and participates in the evaluation and treatment of individuals of all ages with birth defects, intellectual disabilities and other genetic disorders with a particular interest in connective tissue disorders and craniofacial anomalies.

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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