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.

BioMarin presented results of annualized growth velocity in children with achondroplasia who started treatment during adolescence with VOXZOGO. 

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.

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.

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.

Young adults (18-25 years) with a skeletal dysplasia diagnosis and short stature are invited to participate in this research study that aims to understand what independence means to you, and to understand your priorities in terms of independence.