- Looking for drugs to treat myotonic dystrophy Prof Brook, based at the University of Nottingham, completed a two year project where he and his colleagues developed a cell-based drug screen for myotonic dystrophy.
- Investigating different types of muscle stem cells Dr Zammit at Kings College London investigated the function of muscle stem cells to understand more about whether they could be used as a therapy in the future.
- New causative genes discovered for the dystroglycanopathies Prof Muntoni has gathered important information about the genes that are known to be involved in the dystroglycanopathies but also on new genes that are now thought to be involved.
- A new gene causing congenital myasthenic syndromes discovered and an effective treatment found Prof Beeson and colleagues at the University of Oxford uncovered a new genetic fault that can cause congenital myasthenic syndromes as well as a potential new treatment.
- Using MRI to detect changes in the muscles of boys with Duchenne muscular dystrophy Prof Straub has used MRI to gather vital information about how to monitor the changes in the muscles of boys with Duchenne muscular dystrophy for clinical trials.
- First clinical trial for Charcot-Marie-Tooth disease Dr Reilly and her colleagues ran this first clinical trial for Charcot-Marie-Tooth Disease type 1A to see if vitamin C could slow the progression of the disease.
- Improving the delivery of ‘molecular patches’ to muscles The results of this study into exon skipping will help to inform future clinical trials and improve the delivery of the molecular patches to the whole body.
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