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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"

Updates on BioMarin clinical development programme

BioMarin shared an update on the 111-209: Phase 2 Study for Children at Risk of Requiring Cervicomedullary Decompression Surgery and the publication of Phase 3 clinical study.

Pfizer starts phase 1 study with Recifercept

Phase 1 Study to Evaluate The Pharmacokinetics, Safety, And Tolerability Of Single Or Multiple Subcutaneous Doses of Recifercept is now active.

Vosoritide phase 3 clinical trial data have been published

Results from the phase 3 clinical trial for vosoritide, that involved children with achondroplasia with ages between 5 to 18 years have been published.

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
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