We supported the most recent in a long line of Translational Research annual conferences, working in partnership with the International Centre for Genomic Medicine in Neuromuscular Diseases. These conferences focus on neuromuscular research and are an excellent opportunity for researchers to network and hear about the latest trends in research in the neuromuscular field.
17th UK Neuromuscular Translational Research Conference update

The Translational Research Conference is a UK-based meeting. This year it was held in the beautiful surroundings of Goodenough College in central London.
The conference bought together over 180 participants from across the world, including many researchers we’ve funded/are funding. Over the two days we heard 36 talks and saw about 60 poster presentations covering many of the most current topics in the field of neuromuscular disease research.
Members of the charity attending included our CEO Catherine Woodhead, who gave a well-received update on our charitable and research-funding activities. John Copier our Research Manager attending for the first time said:
“I had no idea how brilliant this event would be. I’ve met so many people over these two days. The real power of this conference is that it really gives researchers, particularly those in the UK, a chance to connect and forge collaborations”
Over the past decade, gene therapy has become an important option for treating a range of genetic conditions. As understanding of the technology improves, researchers are expanding the range of conditions for which it is appropriate, and we heard talks on gene therapy for hereditary sensory neuropathy and LAMA2 congenital muscular dystrophy.
There were also a number of talks on antisense oligonucleotide (ASO), also known as molecular patch, therapies. Dr Aurelie Goyenvalle from the French National Institute for Health and Medical Research (INSERM) spoke about the status of these drugs with particular reference to their use in the treatment of Duchenne muscular dystrophy (DMD). Her talk demonstrated how this class of drugs is now being researched for other conditions, like FSHD and type 1 congenital myotonic dystrophy. Importantly, ASOs continue to be another promising approach to the treatment of genetic conditions.
A number of talks focused on inflammatory conditions. It was encouraging to see a range of emerging drugs showing promise in clinical trials for the treatment of inflammatory myopathies and myasthenia gravis.
Professor John Vissing gave the Victor Dubowitz Lecture. Victor Dubowitz, now an emeritus professor, was in attendance. The talk described how Professor Dubowitz made substantial contributions to understanding how exercise affects people with muscle wasting conditions. Professor Vissing built on this, taking about the work he’s done in Becker muscular dystrophy. This was followed by Dr Gita Ramdharry’s talk who further stressed the need to understand exercise and, in particular, how treatments like gene therapy might change the way people with muscle wasting conditions experience exercise. She also touched on some of the work carried out by Dr Louie Lee, whose PhD studentship we funded.
It was interesting to see a number of researchers talking about brain involvement in muscle wasting conditions. This is an under-researched area that affects people with DMD. Researchers presented findings that showed a better understanding of the different forms, or variants, of dystrophin found in the brain and spoke of new approaches to delivering ASOs to the central nervous system. Clinically, this concept is becoming more recognised. Some research showed how better assessment protocols are leading to an understanding that boys with DMD may experience anxiety and problems with the regulation of mood.
The most inventive talk of the conference was undoubtedly by James Alix, who gave his three-minute poster presentation, about the use of a specific method for assessment of muscle health called Raman spectroscopy, entirely in rhyme. We think he can be forgiven for not answering questions in rhyme as well.
We funded a prize for the best poster. This was judged by an independent panel of researchers and was awarded to Jessica Stoodley, a PhD student at the University of Oxford. The prize of £500 will allow Jessica to attend a conference in the next year. We have been supporting Jessica in her PhD studies which look at improving the delivery of molecular patches for spinal muscular atrophy.
A special thank you to the sponsors of this event Sarepta Therapeutics, Pfizer, ITF Pharma UK, Lupin Healthcare and Dyne Pharmaceuticals, without whom the event would not have been possible.
The listed companies sponsored and provided financial support towards the 2024 UK Translational Research Conference but were not involved in the content provided throughout the event.
