BTS endorses new recommendations to protect respiratory health in people with Duchenne muscular dystrophy

New recommendations to protect respiratory health in people with Duchenne muscular dystrophy (DMD) have been published.

They have been developed by DMD Care UK, and project run jointly by Duchenne UK and the John Walton Muscular Dystrophy Research Centre at Newcastle University. The clinical guidelines and patient guide give patients and healthcare professionals information about the best standards of respiratory care in DMD.

The guidance has been endorsed by the British Thoracic Society (BTS) and published in the journal Thorax.

Need for the guidance

DMD is a progressive genetic muscle-wasting disease that affects about 2,500 people in the UK. As people with DMD get older they are at risk of respiratory issues due to the lack of dystrophin, a protein that protects the muscles, including the diaphragm and muscles that control breathing and coughing.

Due to the rarity of the disease, standards of care vary across the country – a problem which the DMD Care UK project is aiming to solve by launching new clinical recommendations and care guides for patients and healthcare professionals.

How the guidance can improve respiratory care for people with DMD

Currently, there is no medication to prevent respiratory muscle weakness but there are vital steps that can be taken to delay it, including starting routine checks from around six years of age.

The new guidelines explain how early monitoring and ongoing management help to keep the respiratory system healthy for as long as possible. They also explain some of the important things that need to be understood by clinicians treating a person with DMD in an emergency or during an illness.

Developing the guidance

DMD Care UK aims to improve care for all people with DMD. The new clinical recommendations and guidelines for respiratory care were produced by DMD Care UK’s Respiratory Working Group, which is chaired by Dr Anne-Marie Childs, Consultant Paediatric Neurologist at Leeds Teaching Hospitals NHS Trust.

Dr Ben Messer, a consultant in Home Ventilation and Critical Care Medicine and clinical lead for the North East Assisted Ventilation Service, was the British Thoracic Society representative on the Working Group.

He explains how the guidelines can help improve respiratory care for people with DMD:

“As patients with Duchenne muscular dystrophy are living longer, they are increasingly likely to present to acute services including respiratory medicine and critical care medicine. This document highlights critical management points during an acute admission. It also details the general respiratory management and the important triggers for referral to complex home ventilation services for further assessment and follow-up.

“The document benefits from multi-professional input as well as patient representation and is a pragmatic guide to the long-term and acute respiratory management of patients with Duchenne muscular dystrophy.”

Based on these recommendations, a new guide was produced for families in collaboration with the patient community.

Phillippa Farrant, a Patient Representative who worked on the new patient guide and clinical recommendations, said:

“As the parent of a son who had Duchenne and having had to battle through the myriad of issues around respiratory care, I felt it important to reflect the patient voice and lived experience when devising the new recommendations and care guide. It’s important to get it right to make the lives of those in the Duchenne community easier.”

Respiratory care guide for DMD patients

The leaflet, and other DMD Care UK guides on bone health, cardiac care, puberty and testosterone, and adrenal insufficiency, can be downloaded from the DMD Care UK website: dmdcareuk.org

- Ends -

Media enquiries

• Journalists can contact Una Farrell, Head of Communications at Duchenne UK, at una@duchenneuk.org or on 07460 256 143.

Notes to editors

• Duchenne UK is a charity that was set up in 2012 by Emily Reuben and Alex Johnson following both of their sons being diagnosed with DMD. They set up Duchenne UK to tackle some of the big challenges in drug development in their search to accelerate the development of treatments. In 11 years, Duchenne UK has raised more than £27 million and used this money to:  
• Fund clinical trials of medicines that are now showing promise for DMD
• Set up DMD Care UK, a national care programme for patients with DMD to stop patients with DMD dying too young because they were not getting the right care. The programme is establishing best practice across all the disciplines involved in DMD care, and works to ensure all medical professionals and parents know exactly what treatment children and adults with DMD need.
• Create a DMD medical research hub with hospital sites across the country, which has led to more trials for DMD treatments than ever before. 
• Develop innovative technologies, the SMART Suit and Dream Chair, to support the independence of people with DMD. 
• You can find out more about the disease and the work of Duchenne UK at org