Clinical Recommendations
The Duchenne Care UK clinical recommendations for Duchenne muscular dystrophy care are evidence-based and consensus-based guidelines for Duchenne muscular dystrophy (DMD) patient care in the UK.
All recommendations are based on the International DMD Care Considerations 2018 published in the Lancet Neurology but are more specific and relevant for the UK healthcare system.
Why use these recommendations
Standards of care improve outcomes and reduce variation. These recommendations have been developed by expert UK clinicians together with patient representatives, through evidence evaluation and consensus building. They have been endorsed by relevant professional bodies and many published in peer-reviewed journals. They will help you to monitor complications, plan proactively and provide clear advice.
Bone and endocrine
These recommendations were drafted by the Bone and Endocrine working group of the Duchenne Care UK project. They have been agreed through consultation and input from North Star specialists from across the UK network, members of the British Paediatric and Adolescent Bone Group and have been endorsed by the British Society for Paediatric Endocrinology and Diabetes (BSPED).
Cardiac care
These recommendations were drafted by the cardiology working group of the DMD Care UK project. They have been agreed through consultation and input from North Star specialists from across the UK network and Duchenne patient and caregiver representatives, and have been endorsed by the British Cardiovascular Society. The recommendations were published in the BMJ Open Heart in October 2022.
Read the recommendations in the BMJ Open Heart or download a PDF copy below.
Corticosteroids
These guidelines refer to the use of corticosteroids (deflazacort, prednisolone and vamorolone) in people with Duchenne. While some of the information might also apply to rare females with Duchenne treated with corticosteroids, the lack of evidence in this population makes the development of any specific guidance very difficult.
Orthopaedic care
Duchenne Care UK's orthopaedic working group has developed a guideline by consensus to inform all those involved in the orthopaedic management of people with Duchenne about the care needs and imperatives. This covers children and adults and focuses on fracture management and elective orthopaedic procedures. The guideline has been endorsed by the British Society for Children's Orthopaedic Surgery.
Physiotherapy and occupational therapy
These guidelines have been developed by Dr Anna Mayhew, Consultant Physiotherapist at the John Walton Muscular Dystrophy Research Centre, Newcastle University, and Duchenne Care UK’s physiotherapy and occupational therapy working group. They have been endorsed by the paediatric and adult North Star Networks collectively. Many of the Network physiotherapists formed part of the Therapy Working Group and all paediatric North Star Network physiotherapists were provided with the opportunity to input the guidelines, and their content. These guidelines are also endorsed by the Neuromuscular Group of Association of Paediatric Chartered Physiotherapists.
Psychosocial care
These recommendations have been developed by the Duchenne Care UK Psychosocial Working Group in collaboration with the wider Duchenne Care UK team. They reflect the expertise of North Star specialists from across the UK, as well as the lived experiences and perspectives of individuals with Duchenne muscular dystrophy and their families.
The guidance is available in three volumes, tailored to different professional audiences: Volume 1: Neuromuscular Teams, Volume 2: Mental Health Professionals, and Volume 3: Education Professionals.
Respiratory care
These recommendations were drafted by the respiratory working group of the Duchenne Care UK project. They have been agreed through consultation and input from North Star specialists from across the UK network and Duchenne patient and caregiver representatives, and have been endorsed by the British Thoracic Society. The recommendations were published in the journal Thorax in December 2023.
Read the guidelines in Thorax or download a PDF copy below.
Vamorolone for Duchenne: a summary of evidence
Prepared by Michela Guglieri together with members of the Corticosteroid Working Group and reviewed by the Family Focus Group of Duchenne Care UK.
How Duchenne Care UK Clinical Recommendations are developed
Written by experts
Written by expert working groups of UK clinicians and patient representatives
Standards of Care
They make global Standards of Care practical for the UK system.
Recognised by NICE
NICE has highlighted the recommendations as a useful resource for clinicians
Accessible
Free, accessible versions for people with Duchenne and their families.
