KXAN

New Data to be Presented for BioMarin's VOXZOGO® (vosoritide) in Children with Hypochondroplasia and Achondroplasia at the American College of Medical Genetics and Genomics ...

Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia. The annualized growth velocity (AGV) increased from 5.12 ...
Healio

Oral achondroplasia therapy receives FDA breakthrough therapy designation

Please provide your email address to receive an email when new articles are posted on . Infigratinib increased annualized height velocity for children with achondroplasia in a phase 2 trial. BridgeBio ...
Seeking Alpha

BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of VOXZOGO® (vosoritide) in Children with Achondroplasia at the European Society for Paediatric ...

SAN RAFAEL, Calif., Nov. 16, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive and consistent results from multiple real-world evidence studies of VOXZOGO ® ...
manilatimes

Results of Pivotal ApproaCH Trial of TransCon® CNP (Navepegritide) in Children with Achondroplasia Published in JAMA Pediatrics

COPENHAGEN, Denmark, Nov. 17, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH ...
Healio

Once-weekly agent boosts annualized growth velocity for children with achondroplasia

A once-weekly investigational prodrug of C-type natriuretic peptide induced a greater increase in annualized growth velocity for children with achondroplasia compared with placebo, according to trial ...