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This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused.
This study looks to assess how celiac disease affects the quality of life of patients between the ages of 13-18.
The Harmony Valve study is for patients in need of pulmonary valve replacement.
Children (less than 10 years of age) are eligible if they have been diagnosed with either a medulloblastoma tumor or CNS embryonal tumors of the brain or spinal cord.
This study wants to determine if racial differences in children that snore are related to factors that can be changed, such as child sleep habits, family factors, and provider unintentional racial bias.
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 research study is to better understand how HIV affects a person's thinking, memories, language, and brain growth.
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).