Participants can access this live virtual event on Monday, October 5, from 7:00 – 8:30 PM CDT.
Achondroplasia (Ach), the most common form of short limb dwarfism in humans, affects 250,000 people worldwide. Although short stature with rhizomelic limb-shortening and macrocephaly are hallmark features of Ach, there are a number of medical complications associated with Ach that significantly increase morbidity and mortality in these patients. Treatment of the developmental complications of Ach currently involves symptomatic management, surgical intervention, and lifelong follow-up care. New therapeutic approaches targeting elements in molecular disease mechanisms of Ach are now in development. These novel investigative agents aim to restore bone growth and prevent many of the medical conditions associated with Ach.
In this virtual live symposium, an expert panel will address the burden of disease and complications associated with Ach, benefits and limitations of current care recommendations, emerging therapies for Ach, and strategies for coordination of care with specialists.