DMD care standards
Introduction
A major international consensus document setting out best practice in care for Duchenne muscular dystrophy (DMD) was published in the Lancet Neurology journal in January and February 2010. The product of an extensive review process by 84 international experts in DMD diagnosis and care, this document is a unique guide to expert recommendations on the care that all individuals with DMD should receive.
The document is available in the January and February editions of the printed journal, online from the journal's website, or here on this page, with special permission from the journal's publisher Elsevier.
 Click here to download the full document (parts 1 and 2 combined)* |
Article details:The Diagnosis and Management of Duchenne Muscular Dystrophy |
Links to the same article via ScienceDirect (only accessible with a subscription to the journal):
Part 1: diagnosis, and pharmacological and psychosocial management*
Lancet Neurol. 2010 Jan; 9(1):77-93. Epub 2009 Nov 27.
PMID: 19945913, DOI: 10.1016/S1474-4422(09)70271-6
Part 2: implementation of multidisciplinary care*
Lancet Neurol. 2010 Feb; 9(2):177-189. Epub 2009 Nov 30.
PMID: 19945914, DOI: 10.1016/S1474-4422(09)70272-8
* NOTE: if you downloaded your copy of the second article BEFORE Jan 18th 2010, please download it again! The new version contains an important correction.
About the document
The drafting of these guidelines was led by the US Centers for Disease Control (CDC) using a rigorous review process (known as the RAND/UCLA Appropriateness Method or RAM) that required experts to rate interventions and assessments used in the management of DMD for “appropriateness” or “necessity” at different stages of the condition. In total the expert panels considered more than 70 thousand different clinical scenarios.
In close collaboration with patient advocacy groups worldwide, the full academic publication has also been transformed into a comprehensive “family guide”. Patients and advocacy groups interested in receiving copies of the family guide or translating it into their own language should .
It is recognised that receiving the best care can dramatically improve the quality of life and life expectancy of individuals with DMD, enabling them to lead fulfilling, independent lives into adulthood. The importance of care recommendations such as these therefore cannot be underestimated. The international guidelines, which cover the diagnostics, cardiovascular, neuromuscular, gastroenterology/nutrition, orthopaedic/surgical, psychosocial, rehabilitation and respiratory fields, can be used by doctors, patients and families worldwide as a guide to the treatment that individuals with Duchenne should receive at each stage of the disease. They are also a valuable tool for lobbying at a national level to enable incorporation of these recommendations into national health systems. The guidelines represent real international consensus including both the medical and the patient advocacy perspectives and can be used across the world as a powerful tool to recognize those centres where best practice is already in place and to identify gaps in care.
Most of the recommendations in the document are not for especially expensive or hard-to-obtain treatments, or indeed for care that is not already available in many of the best centres worldwide. What is stressed is the importance of a multidisciplinary approach – the necessity for patients to see specialists in all the fields that are involved in DMD, and for those specialists to talk to one another to ensure a coordinated approach to the care of each individual. With this authoritative document behind them, TREAT-NMD will work with patient advocacy groups, healthcare professionals and health authorities across the world to establish the best ways of implementing these recommendations and ensuring that all individuals with Duchenne have access to best-practice care.
