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Myology 2024 Paris conference update

Our research communications officer, Dr Andrea Gubas, recently attended the Myology 2024 conference in Paris.

Myology 2024 Paris Conference Update
She shares her highlights and experiences from the conference, as well as the latest research updates.

The Myology 2024 conference took place between 22 and 25 April 2024. Over 1,000 researchers from across the world attended, sharing their research findings through presentations, posters, discussions, or networking. I had the pleasure of being in attendance and catching up with researchers and professionals.

Researchers and clinicians, including PhD students, postdoctoral fellows, and senior researchers, gave updates on their work. From understanding the molecular details of what causes a muscle wasting condition and contributes to its progression, and facilitating diagnostics, to identifying and testing new treatments and more.

Various muscle wasting conditions were covered, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), spinal muscular atrophy (SMA), types of limb-girdle muscular dystrophies (LGMD), facioscapulohumeral muscular dystrophy (FSHD), myotubular myopathies (MTM), metabolic conditions such as Pompe disease, neuropathies, titinopathies and many others.

It’s exciting to see how much work is being done to understand these conditions, with the final aim of finding treatments and improving the quality of life of people living with them.

The effect of exercise
Exercise can be challenging for people with muscle wasting conditions and many are concerned that muscles break down during exercise. However, studies are increasingly showing beneficial effects. Professor John Vissing from the University of Copenhagen talked about studies with people living with Becker muscular dystrophy. These studies showed that exercise is not only beneficial for people with BMD who can walk, but also for wheelchair users, as it can increase their cardiovascular fitness as well as providing some improvement to their overall strength.

Genome editing
We often get asked for more details about genome editing approaches to the treatment of muscle wasting conditions, particularly the method known as CRISPR. CRISPR method uses the immune system of certain bacteria to introduce specific changes into our genes of interest. In laboratory studies, CRISPR has shown promising results and researchers are keen to include it in more clinical studies. A few researchers at the conference, including Professors Charles Gersbach from Duke University and Jaques Tremblay from Laval University, spoke about their work with CRISPR and other gene editing methods for conditions such as Duchenne muscular dystrophy.

Daniel Moore, a PhD student in Professor Saverio Tedesco’s laboratory at UCL and the Francis Crick Institute who is funded by us, spoke about his work with laminopathies and the development of CRSIPR-based therapies. It’s truly fascinating to see how far gene editing has come in the last decade, but we need to be sure all these developing treatments are safe for future trials in humans. It will therefore take some time before they can be considered as potential treatments.

Clinical trials are experiments, not treatments
Setting up clinical trials can be a challenging process. One size doesn’t fit all. People with the same condition can experience different symptoms, have different access to care, and experience different levels of progression of their condition. So, one outcome measure isn’t enough. Professor Jordi Diaz Manera from Newcastle University spoke about the best approaches to set these outcome measures for LGMD clinical trials.

But even with the challenges of setting them up, it’s great to see that many clinical trials are currently in progress or being set up, targeting a range of muscle wasting conditions. This is encouraging as we know not all of them succeed − as of 2022, only 10-15% of clinically trialled drugs reach healthcare services and patients. However, at the conference, we heard positive updates from various ongoing trials, including an update from Professor Francesco Muntoni (UCL) about the clinical trial of GNT0004 gene therapy by Genethon. While initial results appear to be encouraging, this trial is still at an early stage ( Phase I/II), and results are only based on five patients. It’s important to remember that clinical trials are experiments to test if a therapy might work, and not final treatments.

The conference was a great opportunity to meet people in person and to catch up with colleagues from the European Neuromuscular Centre (ENMC), CureCMD, Myotubular Trust and Global LGMD Community Advisory Board (CAB). Many researchers we fund attended the conference, with some of them presenting their work, including Daniel Moore, Professor Jordi Diaz Manera, Professor Rita Horvath, Professor Francesco Muntoni, and others.

We thank AFM Telethon for their support that enabled us to attend the Myology 2024 conference.

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