ALLMedicine™ Alport Syndrome Center

Mar 10th, 2023 - R3R01 is investigational small molecule designed to decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney. This is a single arm open-label study enrolling patients in three coh...

Mar 7th, 2023 - Alport syndrome (AS) is a progressive renal disease characterized by hematuria and progressive renal failure. X-linked dominant (XLAS) is the major inheritance form, accounting for almost 80% of the cases, caused by mutations in COL4A5 genes. Klin...

Feb 10th, 2023 - Alport syndrome (AS) is a progressive hereditary glomerular disease with the prevalence 1 in 50,000. AS is caused by pathogenic variants in the COL4A3, COL4A4, and COL4A5 genes encoding type IV collagen α3, α4, and α5 chains, respectively. There a...

Feb 8th, 2023 - This is a multicenter, open-label, 112-week study of sparsentan in approximately 57 pediatric subjects aged ≥1 year to <18 years with selected proteinuric glomerular diseases, divided into 2 populations, defined as follows: Population 1: Subjects ...

Feb 3rd, 2023 - Alport syndrome (AS) is a hereditary chronic kidney disease (CKD) with X-linked, autosomal and digenic patterns of transmission. Sieving dysfunction of the glomerular basement membrane caused by congenitally defective type IV collagen results in p...

Mar 31st, 2012 - We present clinical practice recommendations for the treatment of children with Alport syndrome who are not enrolled in clinical trials. Our goal is to promote early initiation of a standard therapeutic approach that will facilitate assessment of ...

Mar 10th, 2023 - R3R01 is investigational small molecule designed to decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney. This is a single arm open-label study enrolling patients in three coh...

Feb 10th, 2023 - Alport syndrome (AS) is a progressive hereditary glomerular disease with the prevalence 1 in 50,000. AS is caused by pathogenic variants in the COL4A3, COL4A4, and COL4A5 genes encoding type IV collagen α3, α4, and α5 chains, respectively. There a...

Feb 8th, 2023 - This is a multicenter, open-label, 112-week study of sparsentan in approximately 57 pediatric subjects aged ≥1 year to <18 years with selected proteinuric glomerular diseases, divided into 2 populations, defined as follows: Population 1: Subjects ...

Dec 20th, 2022 - This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation. In total, up to 8 parti...

Dec 19th, 2022 - This single-center, prospective, double-blind randomized placebo-controlled trial will evaluate the efficacy and safety of metformin in Chinese children with Alport syndrome who have received (and continue to receive)) ACEi/ARB.

Mar 15th, 2022 - In late February, the US Food and Drug Administration (FDA) declined to grant marketing approval to the novel agent bardoxolone methyl as a treatment for Alport syndrome, which means this rare genetic disease that causes early onset progressive ki...

Feb 28th, 2022 - Note that some links may require registration or subscription. Ukraine is running out of medical oxygen for critically ill people, the World Health Organization warned. (Reuters) "Show this to Putin." -- The AP captured healthcare workers' unsucce...

Dec 9th, 2021 - An emotional FDA advisory panel on Wednesday unanimously rejected bardoxolone methyl as a treatment for chronic kidney disease (CKD) caused by Alport syndrome, a rare genetic disease that typically leads to renal failure. By a tally of 13-0, the C...

Dec 7th, 2021 - FDA staff raised doubts over bardoxolone methyl as a treatment for chronic kidney disease (CKD) caused by Alport syndrome, citing trial-design issues and questioning the drug's efficacy in briefing documents released ahead of a Cardiovascular and ...

Nov 1st, 2018 - Case Report An 11-year-old boy presented with atraumatic thickening of the skin on the bilateral distal and proximal interphalangeal joints of 1 year’s duration. The patient also noted small bumps of unknown duration across the bilateral palms and.