We are delighted to share our findings in a peer reviewed paper published in the World Journal of Hepatology, titled ‘Characteristics of patients with Wilson Disease in the United States: An insurance claims database study.’
#WilsonDisease (WD) is a progressive, potentially fatal degenerative disease affecting the liver and central nervous system. The worldwide prevalence of WD has been estimated to occur in one in 30,000 to 40,000 people.
The retrospective cohort study revealed its findings on patients in the US with WD based on a comprehensive insurance claims database due to the ongoing challenges of collecting large cohorts of patients with rare diseases and the low prevalence of WD.
The study evaluating 5,376 patients who had submitted documented claims and identified as suffering with WD displayed increasing amounts of psychiatric and neurological manifestations over a 5-year follow-up period. The study ultimately concluded that patients treated with WD in the US had an excess early mortality ratio of 2.19 when compared with the US population, with a mean age of death at 52.7 years. These findings indicate that there is a significant unmet medical need for more effective treatments for WD in the US, alongside corroborating data published from France, Sweden, Poland, South Korea and the UK.
The statistics in the infographic further highlight the prevalence and ongoing burden of patients with WD. Read the full paper 📰 here https://bit.ly/4bURpY6.
#AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT
Thank you Sonia VALERO and Vivet Therapeutics for the work you’re doing in developing a potential gene therapy for patients with Wilson disease.