New potential treatment for achondroplasia by the Tsumaki lab & Astellas Pharma Inc.
2020-12-27Research
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.
First children with achondroplasia receive Recifercept
2020-12-16Research
Pfizer released a Press Statement of the first children with achondroplasia receiving Recifercept, initiating the Phase 2 clinical trial
Vosoritide phase 3 clinical trial data have been published
2020-09-30Research
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
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.
Estudio sobre la calidad de vida de los niños con acondroplasia y sus padres
2019-09-08Research
Desde la perspectiva del niño y de los padres hay algunos estudios sobre la calidad de vida de los niños con enfermedades crónicas, pero los resultados son contradictorios o no se muestran consistentes.
