Author(s): Pietrusz A.; Quinlivan R.; Desikan M.; Marini-Bettolo C.
Source: Neuromuscular Disorders; Oct 2019; vol. 29
Publication Date: Oct 2019
Publication Type(s): Conference Abstract
Abstract:In 2002, the Paediatric North Star Network was established in the UK and resulted in the implementation of the standards of care for Duchenne muscular dystrophy (DMD) across the UK. The network developed a prospective natural history database that could be interrogated as an audit tool. Improved care has led to a growing population of adults with Duchenne who are now on corticosteroid treatment. Long term natural history data in this cohort is lacking, although it is known that survival is increased to the fourth decade and beyond. The Adult North Star Network was created to develop a network of specialist centres looking after adults with Duchenne. The network has an estimated number of 500 adults with DMD, although this figure is growing. The aim of the project is to develop a prospective national natural history database for all adult DMD patients living in the UK. Standardised outcome measures have been agreed and data will be collected for each outpatient encounter. Standardising data collection may improve the standard of care across different regions in the UK. Additionally, the development of population-specific outcome measures will facilitate design of clinical trials and better disease monitoring. We present the early stages of this project development and its goals.
Copyright © 2019
Database: EMBASE