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Searching for the genes that help explain differences in sarcoglycanopathies, forms of limb girdle muscular dystrophy.
Professor Volker Straub and his team are investigating the genes that could explain why people with forms of limb girdle muscular dystrophy called sarcoglycanopathies are affected differently, to try and find new targets for treatments.
Using AI to analyse MRI scans to better predict how muscle wasting conditions progress
Jose Verdu Diaz and colleagues at Newcastle University will be comparing MRI scans from different people with muscle wasting conditions to try to develop an online tool that helps doctors compare scans and understand how the condition is likely to progress and improve understanding of rare muscle wasting conditions.
Developing a new type of muscle scan to identify active muscle damage in people with muscle wasting conditions
Professor Jordi Diaz-Manera and his team are developing a new type of muscle scan to identify active muscle damage in people with muscle wasting conditions. This will show whether this new scanning method could be a useful tool for condition monitoring and developing new treatments.
Testing a new treatment approach for spinal muscular atrophy with respiratory distress (SMARD)
Dr Marion Hogg and team are testing a completely new treatment approach for SMARD, based on restoring levels of a natural chemical that helps keep nerve cells healthy. This could lead to the development of new treatments for SMARD.
Investigating metformin as a possible treatment for facioscapulohumeral muscular dystrophy (FSHD)
Professor Peter Zammit and his team are studying how the drug metformin affects muscle cells in people with FSHD, to investigate whether it could be used as a treatment.
Finding new clues to muscle damage in FSHD
Professor Julie Dumonceaux and her team are exploring why some muscles in facioscapulohumeral muscular dystrophy (FSHD) remain healthy while others become weak and damaged. This knowledge could help researchers develop better ways to monitor the condition and identify new treatment approaches.
Developing a tool to estimate the chance of developing spinal fractures in Duchenne muscular dystrophy (DMD)
Dr Jarod Sze Choong Wong and team are developing a tool to estimate the risk of boys with DMD developing spinal fractures. This will help to catch them before a fracture happens, allowing treatment to start earlier and prevent avoidable pain and disability.
Investigating how Carey-Fineman-Ziter Syndrome (CFZS) causes muscle weakness
Dr Massimo Ganassi and his PhD student will create CFZS laboratory models to study how healthy muscle development is altered and how these changes can lead to muscle weakness so that targeted treatments can be developed.
Improving treatments for ADSS1 myopathy
Dr Fabrizio Pertusati and his PhD student are working to improve a new treatment for ADSS1 myopathy.
Understanding a newly discovered cause of limb girdle muscular dystrophy (LGMD)
Professor Volker Straub and his PhD student are working to investigate a gene called LPCAT3 which may be linked to a new form of a muscle wasting condition. Understanding this gene will help develop future possible treatments.
Understanding how many people are living with a muscle wasting condition in the UK
This project will provide the most accurate picture to date of how many people in the UK are living with a muscle wasting condition by analysing anonymous NHS health records. The findings will help plan clinical services, prepare for future treatments and clinical trials, and provide the evidence to show where resources are most needed.
Building better support for families affected by Congenital Myasthenic Syndromes
Sandhya Rai and her team are working to understand and support the counselling needs of patients and families with congenital myasthenic syndromes (CMS) to identify gaps and create a toolkit to improve patient and family experiences.