Professor Paul Maddison at Nottingham University Hospitals NHS Trust is aiming to validate hand grip strength and MRI as clinical measures that accurately reflect the severity and progression of myotonic dystrophy type 1. This will help to facilitate future clinical trials.
What are the aims of the project?
The aim of this current project is to validate a pair of clinical measures that objectively reflect the severity of myotonic dystrophy type 1, as well as the progression of the condition over time. It builds on the findings of Dr Saam Sedehizadeh’s fellowship project, which collected various clinical measurements from 60 people with myotonic dystrophy type 1 over a three-year period. This showed that hand grip strength is the most sensitive functional measure for monitoring progression and severity of the condition.
In this new project, Professor Maddison and his team will continue measuring the hand grip strength of 34 people who took part in the previous study. This will allow the researchers to detect the rate of change in hand grip strength accurately over a longer period of time. Professor Maddison and his team will also use an advanced Magnetic Resonance Imaging (MRI) technique to assess the health of the forearm muscles that control hand grip. Measurements will be collected over three years, extending the follow-up in these 34 people to six years in total.
Why is this research important?
At present, there are some promising treatments being developed for people with myotonic dystrophy type 1. To be able to assess the effect of these new treatments in clinical trials, it is critical that reliable outcome measures are established. This project will help to achieve this by validating hand grip strength and forearm MRI as a pair of complementary measures that reflect the severity of myotonic dystrophy type 1. It will also generate a better understanding of how muscle health and function changes over time in people with the condition.
How will the outcomes of this research benefit people with myotonic dystrophy?
This project will help to facilitate clinical trials for people with myotonic dystrophy type 1. Validating MRI as an accurate measure of muscle health could also have great value as it is non-invasive and may reduce the need for muscle biopsies in future research and clinical practice.
How might this research impact on other neuromuscular conditions?
This project will validate muscle MRI as an accurate method for examining muscle health, making it a useful clinical measure for a range of neuromuscular conditions.
Project leader: Professor Paul Maddison
Institute: Nottingham University Hospitals NHS Trust / Nottingham University
Condition: Myotonic dystrophy type 1
Duration: three years, starting 2016
Total cost: £74,087
Official title: Longitudinal biomarker refinement in myotonic dystrophy type 1 to inform clinical trial design
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Read our factsheet on myotonic dystrophy
This research would not have been possible without individual donations, family fundraising activities, trusts and corporate support for our Myotonic Research Breakthrough Fund.
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