In this phase 2 dose-finding study, we evaluated the safety and efficacy of oral infigratinib in children with achondroplasia between the ages of 3 and 11 years. A total of 72 children were ...

“The goal of this comprehensive resource is not only to share knowledge about the medical impacts of living with achondroplasia, but to provide families with the tools they need to empower ...

The “Dreambird” study will prospectively assess the burden of complications of achondroplasia in approximately 200 children across multiple sites in Europe, Canada and the United States.

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN), Wednesday announced positive data from the CANOPY clinical program evaluating Voxzogo in children with achondroplasia, a rare genetic skeletal ...

Significant Impact on Bone Length, Strength, Proportionality and Health-Related Quality of Life in Achondroplasia Results from an investigator-led analysis of BioMarin's Phase 2 111-205 study ...