The Burden of Duchenne muscular dystrophy

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Publication - 'The Burden of Duchenne muscular dystrophy'

Treat NMD are pleased to announce the publication of ‘The Burden of Duchenne muscular dystrophy' international study.

Significant hidden costs revealed in international study of Duchenne muscular dystrophy.

In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscular dystrophy (DMD) and that there is a considerable financial burden carried by affected families.

The research, published today in the journal Neurology®, was led by Newcastle University (UK) and the Karolinska Institutet (Sweden) and carried out in collaboration with patient registries for DMD in Germany, Italy, the UK and the United States. Funded by GSK, the aim of the study was to estimate the total cost of illness and the economic burden of DMD.

DMD affects one in 3,500 male births every year. The condition is caused by a faulty gene and the main symptom is severe and progressive muscle weakness, leading to falls and difficulty with motor skills. By the age of twelve most patients need a wheelchair, though treatment with steroids can extend walking ability. Ultimately, patients are unable to look after themselves, and are prone to breathing and heart difficulties which usually result in a life expectancy of around 30 years.

The national DMD registries from the global TREAT-NMD network helped identify the patients with DMD in Germany, Italy, UK, and USA. Researchers asked 770 patients and their primary caregivers in Germany (173), Italy (122), the UK (191) and the USA (284) to complete a questionnaire on their experience of living with DMD and the impact this had on the need to access medical care, employment, leisure time and quality of life.

Further information about the study can be found on the TREAT-NMD website.

TREAT-NMD - Burden of Illness Publication - DMD Publication

2nd July 2014

TREAT-NMD Alliance