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This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in ...

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Dravet syndrome (DS) is an epileptic encephalopathy caused by pathogenic variants in the SCN1A gene resulting in medically refractory epilepsy and psychomotor delays. As a pilot study assessing for ...

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The goal of this prospective study is to validate a wearable EEG seizure detection solution compared to video EEG. Subjects that undergo a clinical video EEG are asked to additionally wear a wearable ...

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This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using ...

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Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who ...

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Background: During the first few decades of life, the brain changes dramatically in shape and function. Sleep lets researchers measure these changes. Researchers want to create a database of sleep ...

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This study primarily aims to determine whether patients diagnosed with osteochondritis dissecans (OCD) of the knee experience psychological stress due to their diagnosis and treatment plan. ...

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This research study is a clinical trial of a new combination of drugs as a possible treatment for relapsed/refractory Ewing sarcoma and/or osteosarcoma. * The names of the drugs are: * ...

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This is a prospective observational study using a mobile study platform (app) that is designed for use on Android phones. Study participants will provide baseline demographic and medical information ...

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