Phase 2 Trial of Vosoritide Use in Patients with Hypochondroplasia: A Pharmacokinetic/Pharmacodynamic Analysis
Vosoritide treatment showed improvement in AHV and height SD in children with hypochondroplasia. PK analysis indicates that drug exposure was correlated to global CNP activity as measured by urine cGMP but did not correlate with growth outcomes. More studies are needed to identify specific patient characteristics that can predict response to therapy and clinical outcomes.
Authors
- Yulan Qi
- Kimberly Boucher
- Andrew Dauber (ORCID: https://orcid.org/0000-0003-4890-0262)
- Despoina Galetaki (ORCID: https://orcid.org/0000-0002-8943-0386)
- Niti Dham (ORCID: https://orcid.org/0000-0002-5467-0208)
- Roopa Kanakatti Shankar (ORCID: https://orcid.org/0000-0001-9653-8865)
- Nadia Merchant (ORCID: https://orcid.org/0000-0001-8775-6481)
- Niusha Shafaei
- Raheem Seaforth
- Anqing Zhang (ORCID: https://orcid.org/0000-0003-3043-8014)
Institutions
- Children's National (US)
- George Washington University (US)
- The University of Texas Southwestern Medical Center (US)
- BioMarin (United States) (US)
Publication Details
- Journal
- Hormone Research in Paediatrics
- Published
- 2024-10-18
- DOI
- https://doi.org/10.1159/000542102
- Citations
- 2
- Primary Topic
- Glycogen Storage Diseases and Myoclonus
- Type
- article
- Field-Weighted Citation Impact
- 0.94