Expert Interview
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, ASNDInstitution: 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.
Please describe your clinical practice, in particular with treating achondroplasia patients, and research in the space.
Added By: max_adminHow many patients do you treat with achondroplasia? What treatment options do they have?
Added By: max_adminWhat is the mechanism of action of a C-type natriuretic peptide like vosoritide, how does it regulate bone growth?
Added By: max_adminChange 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_adminAre You Interested In These Questions?
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