Healx pledges $25,000 to AKU Society patient registry campaign
Patient registries play a critical role in the development of treatments for rare diseases – 95% of which are still without an approved therapy today. Knowledge limitations around rare conditions are a huge barrier to treatment development and are compounded by the fact that rare disease patient populations are often small and geographically dispersed.
Registries collect information about individuals who are affected by a specific condition and are a powerful aid to understanding the nature, variations and natural history of a disease. They can also help surface important information regarding treatments and outcomes in order to improve care and quality of life.
This campaign aims to develop and run a global patient registry for alkaptonuria – also known as AKU, or Black Bone Disease. AKU is an extremely rare genetic condition, affecting roughly one in 250,000 people around the world. It inhibits the breakdown of homogentisic acid in the body, which can cause significant damage to the bones, cartilage and tissues of those affected.
Commenting on the campaign, Nick Sireau, CEO and Chairman of the Board of Trustees at the AKU Society, says: “We’re delighted that Healx is supporting our work to build the world’s first registry for AKU, a debilitating rare genetic disease. This will make a significant difference to our quest to ensure all AKU patients everywhere can access existing treatments and help us gather data to develop new and better therapies.”
Nick is often regarded as the inspiration behind Healx. Co-Founders, Dr Tim Guilliams and Dr David Brown, met Nick back in 2014 when he was trying to redevelop a known compound to treat AKU. Recognising the struggle that rare disease patients and their families face when trying to access effective treatments, Tim and Dave set up Healx in order to apply cutting-edge technology to the redevelopment of known compounds for rare diseases.
Dr Bruce Bloom, Chief Collaboration Officer at Healx, also commented on the grant: “The launch of this campaign is a huge win for the rare community, and Healx is proud to offer its support. This registry will help patients and researchers access real-world data that will hopefully contribute to the development of much-needed treatments.”