FDA approves 1st drug to improve growth in children with achondroplasia
2021-11-23News
On 19 November 2021, the U.S. Food and Drug Administration (FDA) approved Voxzogo (vosoritide), developed by BioMarin, for children with achondroplasia from the age of five, when children still have growth potential as their growth plates (epiphyses) are open.
Listen to the ASBMR 2021 Rare Bone Disease Podcast
2021-10-13News
The ASBMR — The American Society for Bone and Mineral Research — 2021 Annual Meeting Podcasts on Rare Bone Diseases are available for listening.
Quality of life in adults with achondroplasia in the US
2021-09-27News
Study on quality of life and Psychiatric illness in adults with achondroplasia in the US
The Kathryn O. and Alan C. Greenberg Center for Skeletal Dysplasias (Hospital for Special Surgery, NY), published a study1 on quality of life (QoL) and diagnosis of psychiatric disorders in a group of adults with achondroplasia in the US. The study consists of the application of 36 questions covering 8 health concepts (see below) and subsequent comparison of the results of thi […]
Parent and HCP Surveys on Clinical Challenges in Managing Achondroplasia in Infancy and Childhood
2022-09-22Research
Ahead of the upcoming workshop on 27th October 2022, 16:30 CET, Clinical Management Challenges in Infants and Young Children with Achondroplasia, EAF is undertaking two surveys to establish the challenges of managing achondroplasia in these age groups: one for parents of children with achondroplasia and one for clinicians.
