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Zorevunersen (STK-001) in Patients With Dravet Syndrome

The purpose of this study is to determine whether an experimental drug called zorevunersen (STK-001) works to reduce the number of seizures for children and adolescents with Dravet syndrome. Dravet syndrome is a severe form ...


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ION582 in Children and Adults with Angelman Syndrome

The purpose of this study is to determine the safety and effectiveness of an experimental drug called ION582 in children and adults with Angelman syndrome. Angelman syndrome is a rare genetic condition resulting in severe developmental ...


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VEGFR2/MET Inhibitor Cabozantinib in Combination With Cytotoxic Chemotherapy for Newly Diagnosed Osteosarcoma

This study focuses on individuals who have a type of bone cancer called osteosarcoma (OST). The purpose of the study is to evaluate how well an experimental drug called cabozantinib works in combination with methotrexate, ...


Other

Atacicept in Multiple Autoimmune Glomerular Diseases

This study focuses on people who have been diagnosed with IgA nephropathy (IgAN), IgA vasculitis with nephritis (IgAVN), primary membranous nephropathy (pMN), or minimal change disease and focal segmental glomerular sclerosis (MCD and FSGS). IgA ...


Other

Torque Teno Virus and Clinical Outcomes in Pediatric Kidney Transplant

This study focuses on children and teens (under 18 years old) who have received or are expected to receive a primary (first), kidney-alone transplant and who have been prescribed certain immunosuppression medications for their transplant. ...