Reading Time: 9 minutes Research into genetic causes of MND has greatly increased over the last few decades and gene changes have since been found to play a role in both inherited and non-inherited (sporadic) forms of the disease. Since the discovery of the first gene change in MND around 30 years ago, researchers have found over 40 gene…
Reading Time: 6 minutes QurAlis, a biotechnology company focused on developing precision medicines for MND and other neurodegenerative diseases, has announced the opening of a clinical trial in the UK. This trial of a gene therapy, called QRL-201, is a phase 1 trial in people with MND and will test the therapy to determine whether it is safe and…
Reading Time: 4 minutes In 2021, the MND Association, the My Name’5 Doddie Foundation and medical research charity LifeArc established a joint fund – the Motor Neuron Disease Translational Fund – to support translational research in motor neurone disease. In January 2023, £1 million was awarded to researchers at University College London (UCL) to progress two promising new therapies,…
Reading Time: 4 minutes Translational Research is the term used to describe studies that are the bridge between fundamental laboratory-based research and the testing of potential therapies (often by pharmaceutical companies) in the clinic. Without the continued journey of research concepts and ideas from all research and development stages from ‘bench to bedside’ it is almost impossible to develop…
Reading Time: 5 minutes Last Thursday marked an exciting milestone in the search for effective therapies for MND as full results from the early-phase 1/2 trial of Biogen’s tofersen antisense oligonucleotide therapy for SOD1-ALS were published in The New England Journal of Medicine. In the same issue, a study investigating the use of microRNA in a viral vector to…
Reading Time: 4 minutes This blog is part of the ‘Highlights from Perth’ collection of articles, where you can read about the content of some of the talks and posters presented at the 30th International Symposium on ALS/MND. All presentations have a code beginning with ‘C’ followed by a number (e.g. C50). This will help you locate the specific…
Reading Time: 2 minutes This article was written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London. Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for patients affected by a specific genetic form of ALS, the most common type of MND. ALS is a…
Reading Time: 4 minutes After its successful premiere in 2017, the University of Oxford organised another meeting of people affected by inherited MND, called ‘Families for the Treatment of Hereditary MND (FaTHoM)’. This turned out to be yet another excellent day where MND clinicians-researchers presented on topics such as genetics of MND, genetic testing and gene therapies. Below you…
Reading Time: 3 minutes Every day there are two sets of talks going on at the same time during the International Symposium. On Saturday morning there was a symmetry to what was being discussed in these parallel sessions. Both were talking about the inherited form of motor neurone disease (MND). One as reported from Sara’s blog was from the…
Reading Time: 5 minutes During the 25th International Symposium on ALS/MND there were two dedicated sessions for researchers to view over 300 posters. These posters varied from brain imaging to therapeutic strategies. But what is a poster? In this blog I’ll explain more about the session, as well as highlight some of my personal favourites. A biomedical or clinical…
