法布里病
表型
临床表型
新生儿筛查
医学
酶替代疗法
疾病
遗传学
生物
内科学
儿科
基因
作者
Allison Paltzer,Allegra Quadri,Carly Rasmussen,Raphael Schiffmann,Joel Charrow,Carlos E. Prada
出处
期刊:
日期:2025-03-28
卷期号:149 (10): 597-603
被引量:3
摘要
These data suggest the variant impacts enzyme levels, but its effect on health outcomes remains unclear. We further characterize clinical and biochemical data on individuals with the p.A143T GLA variant to help provide guidance on its clinical significance.
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