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Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus.
Study focusing on non-invasive MRI procedures to measure biological activity within leg muscles of individuals, including both healthy volunteers and those with mitochondrial disease.
The purpose of this research study is to figure out if a new smartphone app impacts distracted driving, and to learn parent and teen perspectives on the app.
This study aims to support families with buying, cooking, and eating fresh fruits and vegetables at home! The goal of this study is to learn how fruit and vegetable vouchers and virtual cooking classes and resources affect families' experiences with home cooking and food shopping, as well as caregiver mental health.
16BT052; Unrelated And Partially Matched Related Donor PSCT w/ TCR αβ depletion for patients with BMF
The purpose of this research study is to test a new method of cell processing for stem cell transplants with an unrelated donor or partially matched related donor for patients with BMF who do not have a well matched related donor.
The purpose of this research study is to understand the impact of transplant on the quality of life of the entire family including sibling donors, other siblings and parents.
The purpose of this research is to study 18F-L-Fluoro-DOPA (F-DOPA), a tracer, which is used to take pictures of the pancreas.
This is a Phase 1/2b trial for the treatment of patients with either advanced or refractory B cell Acute Lymphoblastic Leukemia or B-lineage lymphoblastic lymphoma.
A Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Subjects With Severe Sickle Cell Disease
The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease.