making muscle wasting history

Standards of Care and Centres of Excellence

Research has conclusively shown that young people living with Duchenne who have access to multidisciplinary expert care are living longer and have an improved quality of life.

A family guide summarising the results of an international consensus, published in Lancet Neurology, on the the "best practice" medical care of Duchenne is now available.

You can download and print your own copy here. This effort was supported by the US Centers for Disease Control and Prevention (CDC), in collaboration with patient advocacy groups and the TREAT-NMD network. At the heart of the care considerations is the need for a multidisciplinary approach with the input from specialists in many different areas.

Action Duchenne strongly advises you to download and print the family guide and take a copy to the next meeting with your consultant to allow you to be actively enagaged with the medical professionals.

New drugs and medicines are under clinical development and this will add to the complexity of managing the severe muscle wasting and other complications associated with Duchenne. Action Duchenne remains committed to developing new drugs and making sure we have expert clinical management at all stages, including older young men living with Duchenne.

 In January and February 2010 the Lancet Neurology journal published the full internationally agreed Standards of Care for Duchenne: 'The Diagnosis and Management of Duchenne Muscular Dystrophy, part 1 and 2 '. See the TREAT-NMD website for the full article (). Action Duchenne advises that you take these recomendations to your clinical team and check to see if the care you are receiving meets these standards.

The figure has been taken from the Lancet Neurology paper (Nov 2009), part 1: diagnosis, and pharmacological and psychosocial management. It shows the complexity of medical care required for Duchenne patients and also the need for multidisciplinary care.

Do you have access to a Centre of Excellence?

Action Duchenne carried out a consultation on the standards of care received by Duchenne families in the UK between January and March 2009.  The consultation highlights the current disparity between care provision for Duchenne between regions of the UK (essentially a 'postcode lottery').  The consultation calls for the re-organisation of care for Duchenne families and recommends that all families have access to a Centre of Excellence (a one-stop clinic, ideally attached to a research facility) which comprises a multidisciplinary team Figure 1 led by a neuromuscular consultant.  The centres would give Duchenne patients access to expert genetic diagnosis, cardiac assessment and treatment, respiratory management, physiotherapy, occupational therapy, an endocrinologist, a clinical psychologist and other key consultants with Duchenne experience and expertise.  It is vital to ensure that all Duchenne families are treated in line with best practice medical care guidelines.  Currently only Newcastle upon Tyne and Great Ormond Street Hospital offer care that is similar to that recommended by the consultation.

The Walton report published in 2009 also addressed the access to specialist, multi-disciplinary care for people living with muscular dystrophy and related neuromuscular conditions. The All Party Parliamentary Group (APPG) had concerns that the existing specialist services in the four leading neuromuscular centres (Newcastle, Oswestry, Oxford and London) were vulnerable through reliance on charitable funding. In other regions the services can be very poor, with a lack of adult services and weak transition support. The All Party Parliamentary Group (APPG) suggested that it was the responsibility of the Department of Health and the NHS to address these problems.

It has been highlighted by the Walton report that there is a need for individualised specialist care for neuromuscular diseases to be commissioned by one group on a national level, and therefore supports the call for the National Commissioning Group (NCG) and National Specialised Commissioning Group (NSCG) to be dissolved and formed into one advisory group, the National Commissioning Advisory Group (NCAG).  ( www.muscular-dystrophy.org/walton)