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 […]
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“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. […]
“If you have ever put a dollar in a boot or written your name on a shamrock at a gas station, you may have supported the MDA and we appreciate everyone who has done that. The MDA is an umbrella organization that supports 43 different disorders that are all progressive, neuromuscular diseases for which there […]