ALLMedicine™ Alport Syndrome Center

Nov 22nd, 2022 - Alport syndrome is an inherited disease characterized by progressive loss of kidney function. We aimed to evaluate the safety and efficacy of bardoxolone methyl in patients with Alport syndrome. We randomly assigned patients with Alport syndrome, ...

Nov 22nd, 2022 - Bardoxolone Methyl for Alport Syndrome: Opportunities and Challenges.|2022|Quinlan C,Jayasinghe K,|

Nov 14th, 2022 - The evident genotype-phenotype correlation shown by the X-linked Alport syndrome warrants the assessment of the impact of identified gene variants on aberrant splicing. We previously reported that single nucleotide variants (SNVs) in the last nucl...

Nov 10th, 2022 - Data to guide the evaluation of living-related donor candidates for kidney transplant recipients with Alport syndrome (AS) spectrum are limited. We aimed to examine a cohort of living-related donors to recipients with AS and compare their outcomes...

Oct 22nd, 2022 - 34-year-old-female with a medical history significant for Alport's syndrome, chronic kidney disease on dialysis, and hypertension, was brought to the emergency department for sudden onset aphasia and facial droop that began 30 min prior to arrival...

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 ...

Sep 26th, 2022 - 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 ...

Aug 18th, 2022 - 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...

Aug 11th, 2022 - The NKF Patient Network is a longitudinal prospective and retrospective observational cohort study of patient-entered data that collaborates with health systems to obtain additional electronic health records (EHR) data. The NKF also partnered with...

Jul 26th, 2022 - The AFFINITY Study is a phase 2, open-label, basket study to evaluate the efficacy and safety of atrasentan in patients with proteinuric glomerular disease who are at risk of progressive loss of renal function. Cohorts will consist of patients wit...

Jul 7th, 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...

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.