Lee J E, Falk R E, Ng W G, Donnell G N
Am J Dis Child. 1985 Jan;139(1):57-9. doi: 10.1001/archpedi.1985.02140030059029.
We studied two cases of beta-glucuronidase deficiency. One patient's disease was present at birth and the other patient's disease appeared in early childhood. The symptoms observed in both patients, although of differing severity, included peculiar facies, cloudy cornea, hepatosplenomegaly, hernia, kyphosis, recurrent infections, short stature, and developmental delay, as well as increased excretion of urinary chondroitin sulfate A/C and decreased levels of beta-glucuronidase activity. We reviewed all of the reported cases and examined the biochemical and clinical heterogeneity observed in this disorder.
我们研究了两例β-葡萄糖醛酸酶缺乏症患者。一名患者出生时即患有该病,另一名患者在幼儿期发病。两名患者所观察到的症状尽管严重程度不同,但都包括特殊面容、角膜混浊、肝脾肿大、疝气、脊柱后凸、反复感染、身材矮小和发育迟缓,以及尿中硫酸软骨素A/C排泄增加和β-葡萄糖醛酸酶活性水平降低。我们回顾了所有已报道的病例,并研究了该疾病中观察到的生化和临床异质性。