Care Guides Duchenne Muscular Dystrophy

Clinicians
Patients

Clinicians

The Diagnosis & Management of DMD

It is well-understood that receiving the best care can dramatically improve the quality of life and life expectancy of individuals with Duchenne muscular dystrophy, enabling them to lead fulfilling, independent lives into adulthood. The importance of international care recommendations cannot be underestimated.

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 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 2018, significant update to these guidelines was published, this time, as three papers in the Lancet Neurology. In particular, these address three new topics in addition to the eight from 2010.

These international publications 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.

Part 1: Diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Part 2: Respiratory, cardiac, bone health, and orthopaedic management

Part 3: Primary care, emergency management, psychosocial care, and transitions of care across the lifespan

The Imperatives of DMD

PPMD, UPPMD and TREAT-NMD have created a single-page summary of best-practice care in Duchenne muscular dystrophy (DMD). Based on the existing Care Considerations, it will broaden awareness of the “imperatives” in good DMD care, especially amongst non-specialists who are not familiar with the disease and will not have read the full Lancet publication or the condensed care guidelines developed for families.

To disseminate this information as widely as possible, we would be very pleased to hear from anyone who would like to translate the Imperatives into their local language.

PLOS have published an academic paper about the Imperatives of Duchenne. We are delighted that this paper has now also been translated into Spanish.

Please contact info@treat-nmd.com if you can help!