开发了一种新型观察者报告的结果工具 , 作为罕见疾病随机对照试验的主要疗效结果指标。
关键词: Clinical trial Dichloroacetate Observer-reported outcome measure Pyruvate dehydrogenase complex Rare disease
Mesh : Child Child, Preschool Cross-Over Studies Dichloroacetic Acid / administration & dosage Female Humans Male Placebos / administration & dosage Pyruvate Dehydrogenase Complex Deficiency Disease / drug therapy Rare Diseases / drug therapy Symptom Assessment / methods Treatment Outcome
来 源:
DOI:10.1016/j.mito.2017.11.003
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