Guillaume Canaud, MD, PhD, Speaker

‘Prof. Guillaume Canaud is a MD, PhD, head of the Translational Medicine and Targeted Therapy Unit  at hôpital Necker Enfants Malades (Paris)/Université Paris Cité. Guillaume works specifically on rare disorders involving the RAS/PIK3CA/AKT/mTOR pathways. His team develops preclinical models of overgrowth syndromes and vascular anomalies and are specialized in drug repositioning. His group identified a promising therapeutic for patients with a rare genetic disorder called PIK3CA-Related Overgrowth Syndrome (PROS). Guillaume pushed the clinical development of this drug for patient with PROS. Three clinical trials were launched since 2019 and led to the US FDA accelerated approval on April 6th 2022 of alpelisib for patients with PROS aged of at least 2 years old. Guillaume is the recipient of numerous national and international awards for his contribution.’