On the 28th Nov 2018, Ascendis Pharma A/S announced positive preliminary results from a phase 1 trial of TransCon CNP.

Here you will find the highlights of the World Orphan Drug Congress, WODC 2018, where pharmaceutical companies, contract research organizations (CROs) and Health Technology Assessment agencies were heavily represented, with the participation of some patient organizations, discussing pathways related to orphan drug development and clinical trials. The active participation of patients in research was also a key topic.

Bridge Bio has launched a new biotechnology company, QED Therapeutics focused on precision medicine for FGFR-driven diseases, where achondroplasia is included.

Meclozine, a repurposed drug for achondroplasia, is heading for phase I clinical trials. It has been shown to have restored growth rate and bone length in mice, showing promise in the treatment of achondroplasia.

TherAchon is starting a natural history study on Achondroplasia, in preparation for their next clinical trials.

Ascendis Pharma has dosed the first volunteers in a first-in-human phase I clinical trial with TransCon CNP.