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Investigational Therapies for Primary Hyperoxaluria.
Bioconjugate Chemistry ( IF 4.7 ) Pub Date : 2020-06-15 , DOI: 10.1021/acs.bioconjchem.0c00268
Anna Kletzmayr 1 , Mattias E Ivarsson 2 , Jean-Christophe Leroux 1
Affiliation  

Recent years have brought exciting new insights in the field of primary hyperoxaluria (PH), both on a basic research level as well as through the progress of novel therapeutics in clinical development. To date, very few supportive measures are available for patients suffering from PH, which, together with the severity of the disorder, make disease management challenging. Basic and clinical research and development efforts range from correcting the underlying gene mutations, preventing calcium oxalate crystal-induced kidney damage, to the administration of probiotics favoring the intestinal secretion of excess oxalate. In this review, current advances in the development of those strategies are presented and discussed.

中文翻译:

原发性高草酸尿症的治疗方法。

近年来,无论是在基础研究水平还是在临床开发中,随着新疗法的发展,原发性高草酸尿症(PH)领域都带来了令人兴奋的新见解。迄今为止,很少有支持措施可用于患有PH的患者,这与疾病的严重程度一起,使疾病管理面临挑战。基础和临床研究与开发工作的范围从纠正潜在的基因突变,防止草酸钙晶体引起的肾脏损害到给予有利于肠道中草酸过多分泌的益生菌。在本文中,将介绍和讨论这些策略的最新进展。
更新日期:2020-07-15
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