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Kennedy’s Disease vs ALS: How muscle patterns can aid diagnosis and perform as a novel biomarker

Reading Time: 3 minutes Researchers from University College London led by Dr Pietro Fratta and Dr John Thornton found that muscle imaging can help distinguish Amyotrophic Lateral Sclerosis (ALS) from Kennedy’s Disease based on the way specific muscle groups deteriorate in each condition. The method can also help assess the severity of the disease. ALS is a rapidly progressing…

FaTHoM 2: UK-leading MND clinicians on inherited MND

FaTHoM 2: UK-leading MND clinicians on inherited MND

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…

Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial

Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial

Reading Time: 3 minutes A recent press release by the pharmaceutical company Biogen reported preliminary results from an ongoing clinical trial investigating a form of precision therapy in people with SOD1-related MND. This drug, known as tofersen, is now in the final stages of Phase 1/2 testing in centres across the world, including Sheffield in the UK. Tofersen is…

Kennedy’s Disease: focus on muscle damage reveals key biomarker

Kennedy’s Disease: focus on muscle damage reveals key biomarker

Reading Time: 5 minutes Findings from the largest biomarker study of people with Kennedy’s Disease, published in the journal Neurology, found a predictive biomarker to help in differential diagnosis and tracking clinical progression. Led by Dr Pietro Fratta from University College London, the research team highlighted the importance of markers of muscle mass rather than neuronal damage in Kennedy’s…