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.

The purpose of this study is to look at the safety and tolerability (how well you will react) of the study drug (HM15136) and to determine if it is effective for the treatment of Congenital Hyperinsulinism (HI).

This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah™) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL).

A research study about bone health in children and adolescents at high risk of suffering a broken bone.

This purpose of this study is to determine the efficacy of humanized CD19 CAR  T cells (huCART19) in pediatric and young adult patients with high-risk relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).

ICON 3 is a prospective, open label, randomized, multi-center phase 3 clinical trial comparing eltrombopag with the enrolling physician’s choice of front line therapy.

This study is looking to recruit CHOP patients 5-30 years old with suspected/ confirmed bone sarcomas. The study is looking to evaluate a new MRI technique to evaluate bones and any mass in them.

The purpose of this research study is to examine relationships between brain and behavior development in toddlers with and without autism who have older siblings.

In this study, we are developing new magnetic resonance imaging (MRI) and ultrasound methods to determine if it is effective in diagnosing complication in kidney transplant patients.

The purpose of this research study is to collect information over time in juvenile SpA and back pain patients.