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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"

Breaking news - Preliminary Phase 1 data for TransCon CNP

2018-11-30Research
On the 28th Nov 2018, Ascendis Pharma A/S announced positive preliminary results from a phase 1 trial of TransCon CNP.

Report on the World Orphan Drug Congress 2018

2018-11-16Research
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.

QED Tx – a new company working on a treatment for achondroplasia

2018-08-15Research
Bridge Bio has launched a new biotechnology company, QED Therapeutics focused on precision medicine for FGFR-driven diseases, where achondroplasia is included.

Meclozine for achondroplasia heading to clinical trial

2018-07-16Research
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.

A new step forward for TherAchon: the Dreambird study on Achondroplasia

2018-07-13Research
TherAchon is starting a natural history study on Achondroplasia, in preparation for their next clinical trials. Learn more here.

TransCon CNP for achondroplasia starts Phase 1 clinical trial

2018-05-12Research
Ascendis Pharma has dosed the first volunteers in a first-in-human phase I clinical trial with TransCon CNP.
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