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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"

First orphan drug for achondroplasia is validated

The European Medicines Agency (EMA) validated BioMarin's Marketing Authorization Application for Vosoritide to treat Children with Achondroplasia

RIBOMIC starts testing RBM-007 for achondroplasia

RIBOMIC Announced dosing of the first person with RBM-007 in a Phase 1 Clinical Trial for Achondroplasia.

New drug application for achondroplasia by Ribomic

RIBOMIC Announced submission of RBM-007 for Treatment of Achondroplasia

Positive feedback - EMA patient consultation on achondroplasia

In December 2019, EMA prepared a public hearing to evaluate achondroplasia treatment plan. And more than 100 individuals participated.

Public hearing to evaluate achondroplasia treatment plan - EMA

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

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