Treatments available for Duchenne muscular dystrophy - Duchenne UK (original) (raw)
Treatments and care for DMD
Current treatments and therapies can slow down DMD progression and improve quality of life.
We're working to bring more effective treatments to patients by funding research and accelerating drug development, as well as working to improve standards of care in the UK through the DMD Care UK project.
The best care for DMD
Receiving the right treatment and care has a big impact on people with DMD’s quality of life and life expectancy.
Healthcare professionals should refer to The Diagnosis and Management of DMD, an international guide published in the Lancet Neurology in 2010 and updated in 2018 and the DMD Care UK clinical recommendations, which provide more detailed recommendations specific to the UK.
Visit DMD Care UK for more information and resources on the recommended care and treatment for DMD.
Recommended treatments and care for DMD
Other medications and supporting therapies can improve the quality and length of life of those living with DMD and ease the symptoms of the disease. The below recommendations are based on research and clinical expertise and follow the clinical recommendations from DMD Care UK.
Approved medications for DMD
The following medications can be prescribed to treat Duchenne muscular dystrophy and some of the complications of the disease. Not all treatments are suitable for every patient or for every stage of DMD, so speak to your doctor about your/your child's needs.
Different countries have medicine and healthcare regulatory agencies with different criteria for drug approval, so some treatments that have not been approved for use in the UK are available elsewhere. To understand the landscape of treatments currently in clinical trials or approved for use in the USA, visit PPMD's Duchenne Drug Development Pipeline. For treatments in the EU (and some other jurisdictions such as Japan) please visit the WDO's Drug Pipeline.
Decisions to bring treatments available elsewhere to the UK are made by manufacturers. You can find drugs approved in the UK below, and news on drugs in the UK pipeline in our news section.
Find out more
Treatments in the pipeline
Duchenne UK is funding research to bring new effective treatments to patients, faster.
Research we fund
About gene therapy
Gene therapy is a potential treatment for DMD currently being tested in clinical trials.
DMD Care UK
Find out about our project to improve the standards of DMD care throughout the UK.
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Latest news on treatments and therapies
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EMA’s negative decision on Translarna upheld
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New app to prevent the wrong treatments being given to people with DMD in emergencies
Access to treatments, Policy and campaigning
Duchenne UK’s general election manifesto
DMD research
New Duchenne UK funding call for research into transformative treatments for DMD
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Label for DMD gene therapy Elevidys expanded in the US
DMD research
Pfizer gene therapy for DMD doesn’t meet primary or secondary endpoints in Phase 3 clinical trial
Access to treatments
European Commission rejects European Medicines Agency’s recommendation not to renew Translarna
DMD research, News
Duchenne UK and Parent Project Muscular Dystrophy award $500,000 to research into new muscle cell treatment for DMD
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