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Observational outcomes in Japanese children with achondroplasia starting Voxzogo before age 2
jRCT2031240290Recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Dec 2024
- Modality
- Recombinant Proteins and Enzymes, Peptides
- Countries
- 1
01
Overview
Open-label, single-arm trial of vosoritide in achondroplasia (ACH), sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Event rate of ACH-related surgeries of interest. Started Dec 2024; status: recruiting.
02
Drugs
vosoritide
03
Conditions
achondroplasia (ACH)
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