K 通道抑制剂治疗坎图综合征的临床试验准备情况-猫眼课题宝

K 通道抑制剂治疗坎图综合征的临床试验准备情况

批准号：

10053408

负责人：

Dorothy Katherine Grange

金额：

$19.69万

依托单位：

WASHINGTON UNIVERSITY

依托单位国家：

美国

项目类别：

财政年份：

2020

资助国家：

美国

项目状态：

已结题

起止时间：

2020-08-01 至 2022-07-31

来源：

https://reporter.nih.gov/project-details/10053408

关键词：

ABCC9 gene Address Adult Aneurysm Animal Model Attention deficit hyperactivity disorder Birth Blood Vessels Brain Cantu syndrome Cardiovascular Pathology Cardiovascular system Cell model Cells Child Clinic Clinical Clinical Trials Complex Controlled Clinical Trials Defect Development Developmental Delay Disorders Disease Disease Marker Disease Progression Ensure FDA approved Face Fatigue Future Genes Genetic Genetic Diseases Glyburide Goals Heart High birth weight infant Human Hypertrichosis Hypotension Image Investigation Lead Lymphedema Macrocephaly Measures Migraine Molecular Neurologic Non-Insulin-Dependent Diabetes Mellitus Outcome Assessment Outcome Measure Pancreas Patent Ductus Arteriosus Pathologic Pathology Patient Recruitments Patients Pericardial effusion Persistent Fetal Circulation Syndrome Phenotype Polyhydramnios Positioning Attribute Potassium Channel Preparation Pulmonary Hypertension Rare Diseases Readiness Reporting Research Severity of illness Smooth Muscle Myocytes Stroke Sulfonylurea Compounds System Testing Therapeutic Therapy trial Tissues Validation Vascular Smooth Muscle autism spectrum disorder blood glucose regulation channel blockers clinical examination cohort drug testing experimental study gain of function mutation in utero induced pluripotent stem cell inhibitor/antagonist insight neonatal diabetes mellitus neurocognitive test non-invasive imaging operation perinatal complications prenatal recruit

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PROJECT SUMMARY Cantu syndrome (CS) is a rare genetic condition for which there is currently no directed therapy. CS patients suffer from multiple pathologies, but cardiovascular complications, and neurological features are major concerns. CS results from gain-of-function mutations in two specific genes that encode cardiovascular ATP-sensitive (KATP) channels. FDA-approved blockers of these channels are potential treatments. In preparation for a clinical trial, this project will validate cardiovascular and neurological features as markers of disease in a unique cohort of CS patients, and will validate KATP channel inhibitors as appropriate therapy in unique animal models of CS and in patient-derived cells. Successful accomplishment of the project will complete clinical trial readiness for the proposed therapeutic approach.

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Clinical Trial Readiness for K channel inhibitors in Cantu Syndrome

批准号：

10227247
项目类别：
资助金额：

$23.63万
财政年份：

2020
负责人：

Dorothy Katherine Grange
依托单位：

WASHINGTON UNIVERSITY

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