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This study is testing the safety and effectiveness of adding a targeted therapy (ruxolitinib) to standard chemotherapy for children, adolescents, and young adults with high-risk acute lymphoblastic leukemia with CRLF2 and other JAK pathway mutations.
The primary goal of this research study is to learn if patients with autosomal recessive polycystic kidney disease (ARPKD) are at risk for brain aneurysms or cardiovascular abnormalities.
A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease.
Dr. William Gaetz. has partnered with pediatric neurologist, Dr. Eric Marsh and neuropsychologist, Dr. Lisa Blaskey to better understand Epilepsy in children who have an Autism Spectrum Disorder (ASD).
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.
A gene in stem cells will then be edited to help the stem cells make more fetal hemoglobin. Higher levels of fetal hemoglobin can improve red blood cell sickling.
Patients will be followed in the facial nerve paralysis database and will be recruited for completion of a patient-facing survey.
In this study we will test the feasibility of MRI based methods for the assessment of liver disease, and compare the results of these studies to those obtained from clinical liver biopsy.
This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.
Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, may be able to participate.