235 Studies Now Enrolling
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Displaying 1 - 4 of 4
Condition: Pediatrics / Down Syndrome
Investigator: Michael DiLorenzo, MD
Status: Closed
Down syndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Down syndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known to be associated with developmental problems, few studies have explored how CHD can affect the development and behavior of…
Condition: All of Us Research Program - NIH
Investigator: Ali Gharavi, MD
Status: Closed
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…
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
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…
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
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…