
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.
Исследование качества жизни детей с ахондроплазией и их родителей
2019-10-01Research
В августе 2019 года в Orphanet Journal of Rare Diseases1 (европейской базе данных о редких заболеваниях) было опубликовано исследование "Качество жизни детей с ахондроплазией и их родителей – немецкое перекрестное исследование" с целью изучения качества жизни детей с ахондроплазией с точки зрения детей и родителей, а также качества жизни родителей. […]

Voxzogo approved by the European Commission
2021-08-28Medicine
BioMarin announced August 27, 2021, that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide) for the treatment of children with achondroplasia starting at age of 2 years until the closure of growth plates, which occurs after puberty. This treatment is administered once a day by injection.