Our lab is conducting brain imaging research studies investigating people's behavior. We are looking for people between the ages of 18 and 65 to be study participants. As a schizophrenia study participant, you will be asked to complete a set of self-report questionnaires probing personality traits. You will also complete a number of computer-based…
In this study, we are trying to learn more about the symptoms of psychosis, specifically to learn how alterations in the brain may produce certain experiences (including voice hearing or auditory hallucinations) that are common in people with psychosis or schizophrenia. The study involves clinical interviews, computer tests, and magnetic resonance imaging (…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
Our lab is conducting a series of research studies that investigate how specific brain regions are related to functioning in work, school, and in other social situations. As a study participant, you will be asked to complete clinical and neuropsychological assessments, an eye-tracking task, and a non-invasive MRI scan. Study participants earn between $15-50…
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
This study plans to learn more about an investigational new drug called lumateperone tosylate (ITI-007) and may help us find new types of treatment for schizophrenia and schizoaffective disorder.
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
The purpose of this study is to determine whether the brain wave patterns are improved after administration of CVN058 compared to placebo in subjects with schizophrenia. The study uses electroencephalography (EEG) to measure naturally occurring brain waves as you listen to recorded sounds.
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 efficacy, safety and tolerability of different doses and durations of TV-46000 given by subcutaneous (under the skin) injection as compared to placebo for use as a potential maintenance in adults with schizophrenia.
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
In this study, we are trying to learn more about the symptoms of schizophrenia, specifically to learn how abnormalities in the brain may produce certain experiences that are common in patients with schizophrenia. The study involves clinical interviews, clinical chart review, computer tests, and magnetic resonance imaging (MRI) scans.
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…
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…