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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"

Public hearing to evaluate achondroplasia treatment plan - EMA

2019-12-17Research
The Paediatric Committee (PDCO) at the European Medicines Agency (EMA), is currently evaluating the development plans for a proposed treatment for achondroplasia. The PDCO would like to reach out to patients or carers living in EUROPE to gather their experiences of living with achondroplasia.

BioMarin Announces Positive Final Results for Vosoritide - Phase 3

2019-12-16Research
BioMarin announced positive results from the Phase 3 clinical trial for vosoritide (BMN 111) in children with achondroplasia.

Recifercept for Achondroplasia - the new designation

2019-09-30Research
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

2019-09-23Research
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

2019-08-29Research
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

2019-08-28Research
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.
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