News

Reata Pharmaceuticals has a new clinical plan for the approval of bardoxolone methyl in the treatment of chronic kidney disease (CKD) caused by Alport syndrome. Last month, the U.S. Food and Drug Administration (FDA) provided guidance to Reata scientists, suggesting that a single pivotal trial with a…

Analyzing the appearance of the glomerular, using a method called glomerular morphometry, may help to diagnose Alport disease and other kidney conditions in children. The study, “Contribution of glomerular morphometry to the diagnosis of pediatric nephropathies,” published in the journal Saudi Journal of Kidney Diseases and Transplantation, also…

Kidney transplant due to Alport syndrome may be associated with increased production of urine in the first months, according to a case report. This is due to a temporary deficiency in the antidiuretic hormone (ADH) to levels similar to those found in central diabetes insipidus (DI). The study, “Deficiency…

The protein endothelin-1 was found to be linked to both kidney and ear problems in Alport syndrome, according to a mouse study. Targeting endothelin-1 may be a therapeutic approach for the disease, and could prevent hearing loss. The study, “Endothelin-1 mediated induction of extracellular matrix genes in strial marginal cells underlies…

Three-dimensional modeling of kidney structures in a mouse model of Alport disease, using very high-resolution microscopy, revealed how damage to the kidneys evolves. The data provides new insights into disease mechanisms that could lead to more specific treatments for patients with Alport disease, and possibly for those with other types…

Regulus is leveraging its oligonucleotide drug discovery and expertise to develop innovative medicines that target microRNAs for the treatment of several diseases, including Alport syndrome. MicroRNAs are small non-coding RNA molecules that regulate most genes in plants, animals, and some viruses. According to Regulus, a single microRNA can target and regulate hundreds…

Variants in the COL4A3 gene may account for Alport syndrome disease severity in female patients, according to a new study. The study, “Female X-Linked Alport Syndrome With Somatic Mosaicism,” was published in the journal Clinical Experimental Nephrology. Alport syndrome is characterized by chronic kidney disease that progresses to end-stage…

Changes in cellular and molecular signaling, as well as increased mechanical strain, likely contribute to the development and progression of kidney damage in Alport disease. An overview, “Collagen IV diseases: A focus on the glomerular basement membrane in Alport syndrome,” published in the journal Matrix Biology, explored what scientists…

Timely treatment of kidney failure in Alport patients with different mutations can prevent progression to chronic kidney disease and end-stage failure, researchers reported. The study, “Prospective Study On The Potential Of RAAS Blockade To Halt Renal Disease In Alport Syndrome Patients With Heterozygous Mutations,” was published in the journal Pediatric…

Podocyte depletion might be driving the progression of both Alport syndrome and thin glomerular basement membrane disease (TGBM), according to a study published in the scientific journal PLOS One. Therefore measuring podocyte density in kidney biopsies could provide a quantitative readout describing the risk of progression of the conditions event before pathologic changes appear. Moreover, reducing podocyte depletion could have therapeutic effects in Alport syndrome.