HOW CAN WE HELP YOU? Call 1-800-TRY-CHOP
1 - 10 of 33
Research Study for Individuals with Hyperinsulinism, Type 1 Diabetes Mellitus, and Healthy Children
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.
CrCEST MRI in Mitochondrial Disease
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.
AALL1521; Ruxolitinib and Chemotherapy for Patients with JAK-Mutant B-ALL
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.
Aneurysms in ARPKD
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.
Comorbidity of Autism and Epilepsy
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).
EDIT-301: Phase 1/2 Sickle Cell Disease Gene Therapy Trial
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.
Facial Nerve Paralysis Database
Patients will be followed in the facial nerve paralysis database and will be recruited for completion of a patient-facing survey.
Fontan Liver MRE
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.
Gene Therapy Trial for Hemophilia A
This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.