The All of Us Research Program has a simple mission. We want to speed up health research breakthroughs. To do this, we are asking one million people to help us lead the way in discovering better care for all of us. The All of Us Research Program is a large research program. The goal is to help researchers understand more about why people get sick or stay…
This is a study designed for patients with medullary thyroid cancer. Treatment will consist of a drug called Ponatinib. Ponatinib is an investigational agent that blocks abnormal cancer proteins and therefore harms cancer cells. It was recently approved by the Food and Drug Administration for leukemia treatment, but is not approved for medullary thyroid…
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…