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.

The ASBMR — The American Society for Bone and Mineral Research — 2021 Annual Meeting Podcasts on Rare Bone Diseases are available for listening.

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 […]

 

This recent article presents the current and potential pharmacological treatments for achondroplasia, highlighting the advantages and disadvantages of all the drugs that have been demonstrated in human and animal studies in different stages of clinical trials.