PatientsLikeMe, a health data sharing platform that allows patients to manage their own conditions, share their symptoms and learn from others, has created the first open registry for alkaptonuria (AKU).
AKU, a disease that afflicts one in every 250,000 to 500,000 people, inhibits patients’ ability to break down some amino acids that, in turn, build up in tissue and lead to arthritis. According to the National Institutes of Health, this disease can darken urine to “black” and has been associated with heart disease and other health problems. With no cure for this condition, the AKU online registry may be able to aid efforts to develop more effective treatments while providing an accurate figure of how many people have the genetic illness.
Heavy price tags on rare disease drugs can spur a lot of competition between pharmaceutical companies to produce and distribute these medications. As PMLive reports, investigators are studying the use of Swedish Orphan Biovitrum’s tyrosinaemia treatment (Orfadin) for treating AKU, as patients with the disease take the medication off-label because it has yet to be approved.
“This open registry will give anyone—hospitals, pharmacies, providers, nonprofits and patients themselves—a real world view of where and how the disease affects people,” PatientsLikeMe Chairman Jamie Heywood stated.
But more common diseases, such as psoriasis, will soon be using similar methods to encourage drug manufacture. Last year, Merck made an agreement with PatientsLikeMe to collaborate on understanding a much more common disease, psoriasis, with the goal of supporting Merck’s research and development of treatments against the disease.