Duchenne muscular dystrophy materials
The following materials are intended for healthcare professionals. These materials have been developed and funded by PTC Therapeutics.
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International Duchenne Care Consideration infographics
Why a creatine kinase (CK) test?
What to do if you suspect Duchenne muscular dystrophy
The importance of genetic testing
Patient case study – Diagnosis of Duchenne muscular dystrophy (DMD) in an 8-year-old boy
Patient case study – diagnosis of Duchenne muscular dystrophy (DMD) in two brothers
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For more information about Duchenne muscular dystrophy contact us at info@takeonduchenne.eu
International Duchenne Care Considerations summaries
PTC have developed these focused, two-page summaries to provide you with a digestible overview of the 2018 International Duchenne Care Considerations. Click on the summaries to view.
Diagnosis and management of Duchenne muscular dystrophy – Part 1
Diagnosis and management of Duchenne muscular dystrophy – Part 2
Diagnosis and management of Duchenne muscular dystrophy – Part 3
Access the full International Duchenne Care Considerations here.
Pharmacological therapy: Learn more about treatment with glucocorticoids
Along with physiotherapy, treatment with glucocorticoids remains a mainstay of Duchenne muscular dystrophy treatment and it is recommended that individuals should continue taking glucocorticoids after loss of ambulation.1
Recent studies have also demonstrated the benefits of early glucocorticoid treatment, before significant physical decline.1-3
The long-term effects of glucocorticoid therapy have been shown to include*:
Preserved muscle strength and motor function4,5
Delayed loss of ambulation1,4
Preserved pulmonary function1,4,5
Avoidance of scoliosis surgery1,4,5
Improved survival4
*compared to untreated patients
The 2018 International Care Guidelines recommends following an initial consultation with the family, a discussion regarding side effects and a nutritional consultation should occur before any glucocorticoid treatment is initiated.1 Well documented side effects of long-term corticosteroid use can include; weight gain and obesity, acne and warts, cushingoid features, growth retardation and delayed puberty, cataracts, immune/adrenal suppression, glucose intolerance, hypertension, adverse behavioural changes, gastro-oesophageal reflux, peptic ulcer, gastritis, osteoporosis and myoglobinuria.6
Click here for the International Duchenne Care Considerations summaries.
Working together to improve the lives of patients and families
It is possible to help improve the quality of life and life expectancy of individuals with Duchenne muscular dystrophy.1,2 With the right care and access to adequate therapeutic strategies, healthcare teams have been able to:1,3
- Help delay loss of ambulation
- Reduce disease complications
- Improve survival
For your patient, this can mean leading a fulfilling, independent life into adulthood.
These focused, two-page summaries provide you with a digestible overview of the 2018 International Duchenne Care Considerations.
Click on the summaries to view.
Diagnosis and management of Duchenne muscular dystrophy – Part 1
Diagnosis and management of Duchenne muscular dystrophy – Part 2
Diagnosis and management of Duchenne muscular dystrophy – Part 3
Studies have shown that early diagnosis and care can help slow Duchenne muscular dystrophy progression and minimise the risks and complications of the disease.1,2 Click here to find out how you can help drive a successful diagnostic journey.