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Emerging Gene-Targeting Therapies for SOD1-ALS

Emerging Gene-Targeting Therapies for SOD1-ALS

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…

‘Hero’ proteins assemble to stop toxic clumps

‘Hero’ proteins assemble to stop toxic clumps

Reading Time: 5 minutes Much like fictional superheroes who are dedicated to protecting the vulnerable with their supernatural abilities, a newly discovered class of ‘Hero’ proteins with unusual properties can prevent toxic protein aggregation seen in several neurodegenerative disorders, such as MND. This finding may have potential for new treatments and applications in biotechnology.

Lithium revisited: Is there a baby in the bathwater?

Reading Time: 3 minutes At last year’s Airlie House workshop to develop new ALS/MND Clinical Trial Guidelines the focus was, of course, on MND, but there was also important input and learning from outside the field. One of the most fascinating presentations was from an oncologist who was explaining how detailed genetic analysis of tumours was leading to an understanding…

Match-making to find a new treatment for MND

Reading Time: 3 minutes Translating recent scientific discoveries in MND into the development of new and better treatments is a major challenge we face. To facilitate this process within the UK, we’ve recently played a role in match-making a key UK academic neuroscience department and a commercial organisation. Pharmaceutical industry – the big, the small and the CROs Discovery…

X-cell stem cell centre has been investigated by ALS Untangled

Reading Time: 2 minutes With the internet providing an expanse of ‘quackery’ jumbled up with facts, it’s becoming increasingly difficult for anybody to know what source of information can be trusted. This issue is especially apparent to us when we add unproven treatments into the mix. An unproven treatment is, quite literally, a treatment that has no reliable proof for…