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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"

New potential treatment for achondroplasia by the Tsumaki lab & Astellas Pharma Inc.

The Tsumaki lab, at the Center for iPS Cell Research and Application (CiRA), Kyoto University, is leading new research for achondroplasia with ASP5878, an FGFR inhibitor, using iPS cells. This is a study in collaboration with Astellas Pharma.

Future Perspectives on the Treatment of Achondroplasia

Among many rare skeletal disorders, achondroplasia is one of the best known and understood. Yet, its management and treatment are still a major challenge for research centers.

First children with achondroplasia receive Recifercept

Pfizer released a Press Statement of the first children with achondroplasia receiving Recifercept, initiating the Phase 2 clinical trial

Phase 2 Clinical trial with Recifercept starts in December 2020

The Phase 2 Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With Achondroplasia is now live in

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.
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