We've just heard from Dr Laura Ferraiuolo in the Therapeutic Strategies session, where she shared her research on a possible new single dose gene therapy called INS1202. It’s being developed to treat MND caused by a change in the SOD1 gene.  

Her team found INS1202 was safe and well tolerated in animal studies, and successfully reached important parts of the brain and spinal cord. They also found signs it lowered levels of the harmful SOD1 protein, reducing inflammation and helping motor neurones stay healthy and survive.  

These early results are promising and suggest INS1202 is worth exploring as a potential treatment for people with SOD1 MND. 

MND Association Research Fellow shares research into a new target for FUS-MND 

We’ve just stepped out of a session dedicated exclusively to FUS MND. FUS is a protein that can become ‘lost’ in MND and move from the nucleus of the cell, where it should be, to another part called the cytoplasm. That stops it working properly. We heard a brilliant talk from Dr Yiran Wang, one of our Non-Clinical Junior Research Fellows.   

Dr Wang shared the work he and his colleagues have done towards understanding how a common error in the production of the FUS protein might contribute to MND. The team found the mistake, called intron retention, causes common characteristics of MND, and may also be linked to faster disease progression. Their research suggests this error in production of the FUS protein likely plays an important role in the development of MND, and could be a target for treatment.  

In our search for a cure, our community must be front and centre. Every year the Patient Fellows Symposium Programme helps people with MND from all over the world take part in the Symposium. They enjoy a first-hand view of the most recent and promising research and chat to the researchers and healthcare professionals who are bringing hope to the community. 

Just a reminder of our Symposium Stories initiative. We’re keen to show how this event sparks connections and fresh thinking as we all work towards finding effective treatments, and ultimately a cure for MND. You only have to look at the Symposium stories map above to understand the reach of this key gathering of researchers, health professionals and people affected by MND.

Welcome to day three of our International Symposium on ALS/MND, bringing you live news directly from our communications hub in San Diego. 

This morning we’re in the poster hall chatting to researchers from round the world about their science, hopes, plans and vision. 

This morning’s talks kick off with sessions on FUS MND, an area of research we’re currently funding through projects at King’s College London. 

We’ll also be in sessions looking at the role of assistive technology and new developments, and one on techniques to manage symptoms and improve quality of life. 

Another jam-packed agenda awaits...