BioMarin investigational medicine for achondroplasia, BMN-111, known as vosoritide, is currently in phase 3 of the medicine development process and this is an update provided by the company in late July 2019.

The PROPEL trial is the Prospective Study in Children with Achondroplasia now recruiting children from 2,5 to 10 years old.

Dr. Ravi Savarirayan and his team published a paper with results from Biomarin's Vosoritide phase 2 dose-finding and extension study for children with achondroplasia aged 5-14 years, showing an increase in annualized growth velocity.

The Brod Group (TBG), a science-based health outcome research group is developing a study on children’s and parent’s life experience with achondroplasia. This study is a pilot test for a survey and TBG is searching for participants for individual telephone interviews.

ACHieve is the observational study for achondroplasia sponsored by Ascendis Pharma and is now recruiting.

Ascendis Pharma A/S announced on the 28th February 2019, on the rare disease day that the U.S. Food and Drug Administration, FDA, has granted Orphan Drug Designation (ODD) to TransCon CNP.