First orphan drug for achondroplasia is validated
2020-08-14Research
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
2020-07-22Research
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
2020-04-23Research
RIBOMIC Announced submission of RBM-007 for Treatment of Achondroplasia
Positive feedback - EMA patient consultation on achondroplasia
2020-02-04Research
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
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.
