Digging into the recent Phase 2 Cohort 5 results of Bridgebio's ifigratinib in achondroplasiaTicker(s): BBIO, BMRN, ASND
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.
Please describe your clinical practice, in particular with treating achondroplasia patients, and research in the space.Added By: max_admin
How many patients do you treat with achondroplasia? What treatment options do they have?Added By: max_admin
What is the mechanism of action of a C-type natriuretic peptide like vosoritide, how does it regulate bone growth?Added By: max_admin
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?Added By: max_admin
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