The muscles responsible for breathing are absolutely essential for keeping us alive, but we generally don't think about them when everything is working correctly. Diseases that cause muscle weakness can also affect the breathing muscles, resulting in the need for breathing support from a machine and increasing the risk of pneumonia and other serious chest problems. In severe weakness, this can even lead to death.
Brunel University London is involved in an exciting research project investigating gene therapy for young children affected by a rare, life-limiting muscle disease called X-linked Myotubular Myopathy or XLMTM. The gene therapy programme is showing very positive early results, with large improvements in muscle function in those children who have received the treatment (Audentes Press Release). Breathing muscle strength is an important outcome being measured in the gene therapy study. There has however been very little research in the past into what constitutes 'normal' breathing muscle strength in children under the age of six.
This research project will therefore measure breathing muscle strength in a group of young children with no health problems, so that we have an accurate reference point when considering the results from the XLMTM gene therapy trial. As gene therapy offers great promise for a number of serious muscle conditions, the results from this study will also be of use in other research projects in the future. The study will also give us a greater understanding of how the breathing muscles develop and mature in the first few years of life.
Call for participants
We are very keen to hear from parents of children under the age of six with no major health problems. Participating in the study involves a visit to the Brunel University campus at Uxbridge, where we will make simple measurements of breathing muscle strength using a facemask (as shown in this video). The testing should take no more than an hour. There is a second optional visit to repeat the tests (so that we can understand how consistent the measurements are in this age group). Travel costs are paid and all participants will be offered a £40 voucher as a thank you for their time and inconvenience. If you are interested in taking part, or would like more information, please contact the project's lead researcher Emily Knight directly on firstname.lastname@example.org or 01895 268142, or follow the links to our social media pages.