Department of Medicine, Division of Nephrology, Washington University School of Medicine, 4523 Clayton Avenue, St. Louis, MO 63110, USA.
Department of Medicine, Division of Nephrology, Washington University School of Medicine, 4523 Clayton Avenue, St. Louis, MO 63110, USA.
Trends Pharmacol Sci. 2019 Nov;40(11):803-806. doi: 10.1016/j.tips.2019.07.012. Epub 2019 Aug 24.
Alport syndrome (AS), a rare disease of basement membrane type IV collagen, impacts the kidneys, ears, and eyes. In severe cases, kidney failure occurs during adolescence or early adulthood, so most research has focused on remedies for kidney dysfunction. Planned and ongoing clinical studies using targets and therapeutic approaches discussed herein provide new hope for AS patients. The outcomes of these trials could suggest new treatments for more common forms of chronic kidney disease (CKD), demonstrating the importance of focusing on treatments for rare diseases.
Alport 综合征（AS）是一种罕见的基底膜 IV 型胶原蛋白疾病，影响肾脏、耳朵和眼睛。在严重的情况下，肾功能衰竭会在青少年或成年早期发生，因此大多数研究都集中在治疗肾功能障碍上。目前正在进行的临床研究使用了本文讨论的靶点和治疗方法，为 AS 患者带来了新的希望。这些试验的结果可能为更常见的慢性肾脏病（CKD）提供新的治疗方法，这表明关注罕见病治疗的重要性。
