The UK ATTR Amyloidosis Patients’ Association (UKATPA) was founded in 2017 by a group of UK patients with transthyretin (TTR)-type systemic amyloidosis (ATTR), supported by the consultant physicians of the UK NHS National Amyloidosis Centre.
In recent years there have been major advances in the field of ATTR amyloidosis. The processes responsible for formation of TTR amyloid have been elucidated for the first time and intense activity in drug development promises the early advent of potentially effective prophylactic and disease modifying new medicines. Hereditary ATTR amyloidosis, caused by mutations in the TTR gene, is the most common form of hereditary amyloidosis but is nonetheless very rare. However, crucially, there is now compelling evidence that acquired ATTR amyloidosis, caused by normal wild type TTR, is far more common than was previously believed.
In this exciting environment of change and optimism, our members decided to found a UK patient group dedicated to the interests of people in the UK with ATTR amyloidosis and to ensuring their participation in patient care and in disease and therapeutic research. We are building on the work of the existing UK Amyloidosis Advisory Group (UKAAG), which was founded by patients, carers and NAC consultants in 2013 as the UK Patient and Public Involvement (PPI) vehicle for all types of amyloidosis.
Different types of amyloidosis are very different diseases, with very different treatments. The new UKATPA group will focus specifically on PPI in relation to ATTR. We will provide support and information for patients with ATTR amyloidosis, involve the patients in all aspects of management and research, and work closely with the amyloidosis specialist physicians at the NAC to promote interdisciplinary communication with local doctors treating ATTR patients throughout the UK.
Our key objectives will be enhancement of clinical and basic science research into ATTR amyloidosis, facilitation of patient participation in clinical trials of new drugs for ATTR amyloidosis and expediting drug approval processes. When new drugs for ATTR amyloidosis receive licensing approval we will aim to ensure clinically appropriate access for all UK ATTR amyloidosis patients.