Exploratory study of autophagy inducer sirolimus for childhood cerebral adrenoleukodystrophy.
Frontiers in pediatrics（2023）
Abstract
Our study suggested that autophagy inducer sirolimus is safe for CCALD. Sirolimus did not improve clinical symptoms of patients with advanced CCALD significantly. Further study with larger sample size and longer follow-up is needed to confirm the drug efficacy. https://www.chictr.org.cn/historyversionpuben.aspx, identifier ChiCTR1900021288.
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Key words
childhood cerebral adrenoleukodystrophy, autophagy, sirolimus, white matter hyperintensities, demyelination
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