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10 | InTouch SUMMER 2024 RESEARCH NEWS GRASP BMD – defining the endpoints in Becker muscular dystrophy The team at the CBR Neurogenetic Research Clinic are looking for participants with a genetic diagnosis of Becker muscular dystrophy (BMD) to participate in a natural history study called Defining the Endpoints in Becker Muscular Dystrophy. In clinical trials the ‘endpoints’ are the key objective measures that are predicted to show whether the treatment is working. By participating in GRASP BMD you’ll be part of a multi-site international study that aims to include eighty men from sites in the UK, USA, and New Zealand. This study is designed to find better ways to record how Becker muscular dystrophy impacts people over time. We’ll be exploring the natural history of BMD, how the disease progresses over time, and determining the feasibility and suitability of clinical outcome assessments. These analyses will evaluate the responsiveness of the measures, validity, and help define an ideal clinical trial population. Blood biomarkers will also be investigated. The study visits will take place at the CBR Neurogenetic Clinic at the University of Auckland. The first (baseline) visit takes place over two days. Subsequent visits will take just one day and are 12 months apart. All travel and accommodation costs will be covered. As part of each study visit you’ll see a physiotherapist, have your heart checked, see a doctor, complete some questionnaires, and provide a blood sample. Please contact Miriam Rodrigues if you’re interested – email neurogenetics@adhb.govt.nz To speak to us about leaving a gift in your will, please email info@mda.org.nz We have been helping Kiwi families for more than 60 years and by making a bequest, you are ensuring the sustainability of our organisation so that we can continue to be there for generations to come. Any bequest, no matter what size, will directly help those living with muscle wasting neuromuscular conditions, and enable us to continue our work within your community. Your good will benefits families