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New Medication Approved in the United States for Children with Achondroplasia

2026-04-28Research
Recently in 2026, the U.S. Food and Drug Administration (FDA) approved Navepegritide, marketed as Yuviwe by Ascendis Pharma, for the treatment of children with achondroplasia aged 2 years and older who still have open growth plates. Achondroplasia is a genetic condition that affects bone growth and is associated with a range of lifelong medical complications.

Infigratinib: Clinical Trial Results Update for Achondroplasia

2024-10-30Research
QED Therapeutics (an affiliate of BridgeBio) has announced the results of a clinical trial group from the PROPEL 2 Study, a Phase 2 trial of infigratinib for children with achondroplasia.

Voxzogo results for achondroplasia and other growth-related conditions

2024-05-08Research
BioMarin presented results of annualized growth velocity in children with achondroplasia who started treatment during adolescence with VOXZOGO. 

Limb Lengthening in Achondroplasia: Considerations and Experiences

2024-03-21Research
What do you think about Limb lengthening in achondroplasia? If you have not considered it, not done it, or you have, please share your opinion.

TYRA-300 Heading to Clinical Trial in 2024

2023-12-06Research
The investigational drug TYRA-300 demonstrated significant increases in growth and bone length in a mouse model of FGFR3-Related Skeletal Dysplasia on recent study and is now closer to a Phase Clinical Trial.

Movement Patterns Research Study

2023-08-08Research
Take part in this research study that focus on movement patterns! If you accept to participate, you will be asked to fill in a questionnaire and to walk for 15 minutes carrying your mobile phone and using a specific app for movement analysis that will record your movement data while walking.
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