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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"

Report on the World Orphan Drug Congress 2018

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

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

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

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

TransCon CNP for achondroplasia starts Phase 1 clinical trial

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

Study on achondroplasia – request for participation in the USA

The Brod Group, a health outcomes research, and consulting firm are currently recruiting people for an international study of pediatric achondroplasia. The authors aim to get a better understanding of the experiences and daily life of children and adolescents with achondroplasia, as well as the experiences of parents who have children with achondroplasia. Find out more about the study and how to participate here.
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