The US Food and Drug Administration (FDA) has approved ZANVASTRO (zilganersen) for the treatment of Alexander disease (AxD) in paediatric and adult patients, providing the first disease-modifying treatment for this ultra-rare and progressive neurological disorder. Zilganersen is an RNA-targeted medicine designed to reduce production of GFAP, addressing an underlying mechanism of the disease. The 50…
Category: Neurodegenerative diseases
Gut bacteria may reach the brain through the vagus nerve, mouse study suggests
Researchers have identified a potential new pathway by which gut bacteria may enter the brain, providing fresh insight into the gut-brain axis and its possible role in neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease. The study, published in PLOS Biology, found that a short-term high-fat diet disrupted the intestinal barrier in mice, allowing…
UCL Faculty of Brain Sciences researchers awarded UKRI Future Leaders Fellowships
Congratulations to Angelika Zarkali (ACNR’s Conference News Editor), and Mathieu Bourdenx, who have both been awarded UK Research and Innovation Future Leaders Fellowships! Angelika Zarkali, Senior Research Fellow and Honorary Consultant Neurologist at UCL Dementia Research Centre, Queen Square Institute of Neurology, has been awarded a fellowship worth £2.3 million to explore new treatment approaches…
Maura Malpetti awarded European Young Researcher Award 2025
Congratulations to Dr Maura Malpetti (UK DRI at Cambridge), who has won the 2025 European Young Researcher Award from the Fondation Recherche Alzheimer in France. The foundation funds the advancement of research in Alzheimer’s disease and related syndromes in France and beyond. The award supports and encourages an individual European researcher with exceptionally promising work. Maura’s research focuses on in vivo…
Apitegromab shows significant motor gains in SMA patients
Scholar Rock has published pivotal Phase 3 data in The Lancet Neurology demonstrating that apitegromab—a first-in-class muscle-targeted therapy—delivers clinically meaningful motor function improvements in children and adults with spinal muscular atrophy (SMA) receiving SMN-targeted treatment. Study design and population The SAPPHIRE trial enrolled 188 nonambulatory patients aged 2–21 with Type 2 or 3 SMA, all…
More hope for children with SMA?
Biogen’s Spinraza follow-up helps children with SMA who didn’t benefit from Zolgensma Biogen has cited early results from a small phase 1 trial as evidence that its antisense oligonucleotide salanersen could help children with spinal muscular atrophy (SMA) who are still struggling to sit independently despite having received Novartis’ Zolgensma. The phase 1 study was…
Research finds length of gene not linked to PSP or CBD
Research has found there is no link between genetic variations (repeat expansions) of C9orf72 and the development of rare brain diseases such as Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration (CBD). The research, which was led by Dr David Vaughan at UCL and was part funded by UK charity PSPA as part of the PROSPECT-M-UK…
Spinocerebellar ataxia – troriluzole topline results
Biohaven achieves positive results in pivotal study September 23 2024 Biohaven Ltd have announced positive topline results from pivotal Study BHV4157-206-RWE (NCT06529146) demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA…
