236 Studies Now Enrolling
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Displaying 1 - 5 of 5
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: Psychiatric Disorders / ADD/ADHD
Investigator: Jonathan Posner, MD
Status: Closed
In this study, we are trying to learn more about how stimulant medication produces an improvement in emotional lability (frequent mood changes and excessive emotional reactions) in patients with Attention Deficit Hyperactivity Disorder (ADHD.) Although ADHD is primarily associated with inattention, hyperactivity, and impulsivity, emotional ability plays a…
Condition: Psychiatric Disorders / ADD/ADHD
Investigator: Jonathan Posner, MD
Status: Closed
Children with Attention-Deficit/Hyperactivity Disorder (ADHD) are typically treated with two types of medications: stimulants and non-stimulants. These medications work in different ways to treat ADHD symptoms, but we do not fully understand the brain mechanisms underlying the medications' effects. This study will use MRI scanning to look at changes in…
Condition: Cancer / Thyroid Cancer
Investigator: Antonio Fojo, MD, PhD
Status: Closed
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…
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…