Isenberg J N, Sharp H L
J Pediatr. 1975 May;86(5):713-7. doi: 10.1016/s0022-3476(75)80355-6.
A 5-year-old girl with coarse facies, visceromegaly, and vacuolated lymphocytes is presented as the first case of aspartylglucosaminuria diagnosed in this country. This metabolic defect in glycoprotein catabolism can be clinically confused with other storage diseases such as the mucopolysaccharidoses and mucolipidoses. It is not diagnosed by routine laboratory screening methods. Special studies are required to confirm the diagnosis, but a thin-layer chromatography method for screening urine is presented for use when the diagnosis is suspected. The developmental potential in this inborn error of metabolism is documented.
一名5岁女童,面容粗糙、内脏肿大且淋巴细胞有空泡化,是该国诊断出的首例天冬氨酰葡糖胺尿症患者。这种糖蛋白分解代谢中的代谢缺陷在临床上可能会与其他贮积病混淆,如黏多糖贮积症和黏脂贮积症。常规实验室筛查方法无法诊断该病。确诊需要进行特殊检查,但本文介绍了一种用于尿液筛查的薄层色谱法,以便在怀疑诊断时使用。本文记录了这种先天性代谢缺陷患者的发育潜能。