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.1,2

With the right care and access to adequate therapeutic strategies, healthcare teams have been able to:1,3

  • Delay loss of ambulation
  • Reduce disease complications
  • Improve survival


For your patient, this can mean leading a fulfilling, independent life into adulthood.

Read the blog: A parent’s voice

Read a mother’s account of family life after receiving a Duchenne diagnosis for her son.

Never will I forget the sunny day in March, many years ago, when the paediatrician announced: "Your son has a serious, incurable muscle disease." He looked at me cautiously, as if waiting for a reaction. A sign that I understood what he had just said. But there was nothing. My head was fuzzy.

Read on

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YOUR ROLE IN DUCHENNE MANAGEMENT

Duchenne care can be complex.

It requires neuromuscular, respiratory, cardiac, orthopaedic, endocrine, psychosocial and rehabilitative interventions – all of which evolve over the course of the disease.1

As such, international care guidelines for Duchenne recommend a coordinated, multidisciplinary approach to care.1

By assembling a group of providers in a multidisciplinary team, the patient can benefit from:1

  • Integrated skills and knowledge from multiple disciplines
  • A coordinated and comprehensive treatment plan

The Duchenne multidisciplinary team

Click each icon to find out more

Please note that the information in this figure is intended to provide an overview only and is not exhaustive information. Please refer to the international care guidelines and any local guidelines for more information.

Adapted from Birnkrant 2018 part 1; Muscular Dystrophy UK1,4

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WHY IT IS IMPORTANT TO TREAT DUCHENNE

Studies have shown that early diagnosis and care can help slow Duchenne progression and minimise the risks and complications of the disease.1,2,11–13

This means that patients may:1–3,11–13

  • Remain ambulatory for longer
  • Preserve their pulmonary and cardiac function
  • Live longer
  • Have a better quality of life
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THERE ARE TWO MAIN APPROACHES TO DUCHENNE PHARMACOLOGICAL THERAPY

Agents* that help manage the signs and symptoms of Duchenne

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Glucocorticoids for the treatment of muscle strength and function1,14

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Angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) for the treatment of heart disease5

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Bisphosphonate therapy for the treatment of osteoporosis5

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Hormone replacement therapy for the treatment of impaired growth, delayed puberty, and adrenal insufficiency1

*Please note this is not an exhaustive list

Agents that treat the underlying cause of Duchenne

Therapeutic strategies include:*2

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Mutation-specific therapies or gene replacement therapies aiming to restore dystrophin production

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Muscle membrane stabilisation and/or upregulation of compensatory proteins which are structurally and functionally similar to dystrophin

*Some of these treatments have been approved by regulators while others are near, or in, regulatory review and might become available in the future 1,2

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DUCHENNE INTERNATIONAL CARE GUIDELINES RECOMMEND CONSIDERING GLUCOCORTICOIDS FOR ALL PATIENTS1

Treatment with glucocorticoids remains a mainstay of Duchenne 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.1,13,15

The long-term effects of glucocorticoid therapy have been shown to include*:

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Preserved muscle strength and motor function3,14
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Delayed loss of ambulation1,3
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Preserved pulmonary function1,3,14
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Avoidance of scoliosis surgery1,3,14
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Improved survival3

*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 corticosteroid 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.16

For more information, Click here for the International Duchenne Care Considerations summaries.

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Educate patients on the need for disease management and care
Duchenne and You is a patient-friendly website developed and funded by PTC Therapeutics that helps both individuals with Duchenne and their families understand what disease management and care are available and why they are important
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