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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"

QED Therapeutics starts observational study

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

Vosoritide for achondroplasia - Phase 2 data results

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.

Update - Study on achondroplasia

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.

Ascendis starts an observational study for achondroplasia

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

Orphan Drug Designation Granted for TransCon CNP

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.

The Norwegian Adult Achondroplasia Study

The Sunnaas Rehabilitation Hospital is conducting an observational study on the prevalence of medical complications in adults with achondroplasia and their impact on daily activities entitled "The Norwegian Adult Achondroplasia Study".
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