Dr. Sabrina Paganoni, M.D., Ph.D., Investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Justin Klee, Co-Founder and Co-CEO of Amylyx Pharmaceuticals discuss ALS (amyotrophic lateral sclerosis or Lou Gerhig’s disease) and a new investigational treatment that has shown an ability for patients to retain function and independence longer. The findings from that study are now being published in a leading medical journal. ALS as a disease has been notoriously challenging for new drug development, and this represents a historic step forward.
Sabrina Paganoni, MD, Ph.D. Dr. Paganoni is an Assistant Professor at Harvard Medical School and works as a physician scientist at the Healey & AMG Center for ALS at Massachusetts General Hospital and at Spaulding Rehabilitation Hospital. Her research focuses on developing new treatments for ALS (amyotrophic lateral sclerosis, Lou Gehrig’s disease). She designed and is currently leading several ALS clinical trials that include novel endpoints and biomarkers and innovative trial designs, including Amylyx’ CENTAUR clinical trial. She is currently the co-Principal Investigator of the HEALEY ALS Platform Trial, the first platform trial for ALS in the world.