The Brod Group, a health outcomes research, and consulting firm are currently recruiting people for an international study of pediatric achondroplasia. The authors aim to get a better understanding of the experiences and daily life of children and adolescents with achondroplasia, as well as the experiences of parents who have children with achondroplasia. Find out more about the study and how to participate here.

Developing during childhood, obesity is a common complication associated with achondroplasia, affecting more than 50% of people with this condition. Here we present a paper demonstrating that obesity in achondroplasia is markedly different from other kinds of obesity and is less prone to developing most of the complications associated with obesity. The authors have also demonstrated that treatment with soluble FGFR3 reduces weight and normalizes the metabolic disturbances associated with achondr […]

We report on the 2017 International Skeletal Dysplasia Society (ISDS) meeting. Previously unknown metabolic disturbances were discovered in achondroplasia and new results were presented on meclazine for achondroplasia.

BioMarin shared a document with all associations updating everyone who is interested in their upcoming medications on their clinical trials. Read the whole document here.

Therachon is developing a drug for achondroplasia, which has now crossed an important threshold in drug development for rare diseases. It has obtained Orphan Drug designation by the American and European regulatory agencies (FDA and EMA, respectively), taking it a step closer to a possible market authorization.

This is an update on the observational study which collects information to establish a baseline for the other clinical trials by BioMarin. It is necessary to be enrolled in this one first for 6 months. Learn where to enroll here.