Dance is a complex activity involving the integration of rhythm, spatial patterns, synchronization to external stimuli and whole-body coordination. While regular exercise is often recommended for people with Huntington's Disease (HD), there are no studies examining the use of dance to improve balance and mobility and positively affect the quality of…
The DIMENSION Study is evaluating the safety and effect on cognitive symptoms of an investigational oral drug in adults with early Huntington's disease. Cognitive symptoms may include difficulty paying attention or trouble thinking through steps of an activity or complex problems. Symptoms may also include difficulty planning, remembering, and staying…
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…
We are inviting patients with active Rheumatoid Arthritis (RA) to help us better understand how treatment options affect inflammation in soft tissues. With a combination of blood work, a full-body FDG PET-CT scan, questionnaires, and a soft tissue biopsy (from an area around the belly button), we hope to see how RA interacts with these multiple areas of…
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…
The study aims to compare the effect two different RA treatments on cardiovascular health in patients who are considering a treatment change. The study is recruiting patients who are only taking methotrexate and are switching treatment due to a lack of improvement. The trial will randomize participants to take a TNF inhibitor (Enbrel or Humira) in addition…
We are doing a research study to learn more about why people with rheumatoid arthritis (RA) are prone to heart disease. We are recruiting patients diagnosed with Rheumatoid Arthritis without a personal history of heart disease. This study involves one initial full visit as described below, and a brief follow up visit that will take place approximately 2 or…
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 research study is to find out whether a new investigational drug called SRX246, which might be useful to Huntington's Disease (HD) patients who sometimes feel irritable, angry, or even aggressive, is well tolerated and safe when it is given two times a day by mouth at doses as high as 160 mg. As part of this study, we will use a…
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…