Understanding Spinal Muscular Atrophy: A Chief Scientific Officer's Perspective

In the second episode of a three-part series dedicated to highlighting rare disease, Conference Forum reporter Danny McCarthy sits down with C Frank Bennett, PhD, of Ionis Pharmaceuticals, to discuss Spinal Muscular Atrophy. Dr Bennett is Executive Vice President and Chief Scientific Officer at Ionis Pharmaceuticals, and one of the founding members of the company. He is responsible for continuing to advance antisense technology and expanding Ionis drug discovery platform. Dr. Bennet is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen).  Spinal muscular atrophy is a genetic, degenerative neuromuscular disorder characterized by weakness and wasting in muscles used for movement. It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement.  Dr Bennett includes two sources of information about Ionis and SMA: Ionispharma.com and Spinraza.com.