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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"

Recifercept for Achondroplasia - the new designation

Recifercept is the name Pfizer as selected for the orphan drug for achondroplasia. Pfizer acquired Therachon in May 2019, and with that, TA-46, the new molecule being developed as a potential treatment for achondroplasia.

TransCon CNP update - The ACcomplish trial heading to start

Ascendis Pharma is developing the TransCon CNP program at a swift pace and moving forward with phase 2 ACcomplisH Trial to evaluate the safety and efficacy of TransCon CNP in children with achondroplasia.  

Study on Quality of life of children with achondroplasia and their parents

It has been recently published, in August 2019, the "Quality of life of children with achondroplasia and their parents - a German cross-sectional study" at the Orphanet Journal of Rare Diseases. This study investigated the quality of life of children with achondroplasia from children and parent’s perspective and also the parental quality of life.

Update of BioMarin Clinical Development for Achondroplasia

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.

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.
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