SMA – Patient Activation Network

Dr. Susan Begelman on the Patient Activation Network

PAN57: Spinal Muscular Atrophy with Dr. Susan Begelman

Posted on August 13, 2019August 14, 2019 Matt CavalloLeave a commentPosted in Podcast

Spinal Muscular Atrophy (SMA) is a rare disease that affects the motor nerve cells in the spinal cord. SMA may affect a person’s ability to eat, walk, or even, breathe. SMA is also the number one cause of death in infants. In this episode, Dr. Susan Begelman discusses what causes SMA, newborn screening and hope […]

PAN35: MOVR Data Hub with Grace Pavlath, Ph.D.

Posted on April 9, 2018May 1, 2018 Matt CavalloLeave a commentPosted in Podcast

“This is a very interesting time in therapy development. Just in this decade alone there have been seven therapies approved which is unprecedented. If you look at the 70’s, 80’s and 90’s, there was one per decade,” reflected Grace Pavlath, PhD. “If you look at today’s pipeline, there are 250 unique compounds in various stages. […]