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中文摘要
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英文摘要
This study is limited to patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, but has the potential to significantly improve the lives of patients with other forms of adrenal insufficiency. Each of these diseases requires life-long treatment and this novel approach to drug delivery has the potential to significantly improve patients lives. This newly approved pilot study has enrolled one patient and is actively recruiting. In addition, this study will provide information on the safety and tolerability of using a pump to deliver hydrocortisone, and will generate data that will be used in the design of future pediatric studies.
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Modified-release Hydrocortisone Therapy as a Treatment for CAH
Novel treatment approaches: sex steroid blockade in children
  • 批准号:
    10916862
  • 项目类别:
  • 资助金额:
    $0.0万
  • 财政年份:
    --
  • 负责人:
    Deborah Merke
  • 依托单位:
Phase 2 Multi-center Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
  • 批准号:
    10252568
  • 项目类别:
  • 资助金额:
    $0.0万
  • 财政年份:
    --
  • 负责人:
    Deborah Merke
  • 依托单位:
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