The landscape of neurology biomarkers has evolved significantly in recent years, affording new clinical insights that are helping to reshape patient care. During an expert interview conducted by the European Medical Journal (EMJ), Simon Thebault, Assistant Professor at Montreal Neurological Institute, and attending physician at McGill University Health Centre, Canada, explored important advancements in blood-based neurology biomarkers, focusing on the key role of neurofilament light chain (NfL). NfL is a neurone-specific protein and biomarker of axonal damage that can provide novel insights into disease activity in multiple sclerosis (MS) and aid in prognostication. New innovations in blood-based testing for NfL are helping to facilitate the clinical application of this key biomarker to improve MS disease management.

This symposium was held at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference 2025, in Dallas, Texas, USA. Speakers gave an overview of the patient experience and natural history of Becker muscular dystrophy (Becker) and presented the latest Phase I/II clinical data for the novel investigational agent, sevasemten. Becker is a serious, rare, neuromuscular disorder that produces irreversible muscle loss and progressive impairments in mobility and cardiac function, amongst other physical and emotional effects. However, there are currently no approved treatments for Becker. In a powerful start to the symposium, Michael Voto, New Jersey, USA, a patient advocate living with Becker, provided insight into the lived experience of Becker and, together with Abby Bronson, Vice-President of Patient Advocacy, Edgewise Therapeutics, Colorado, USA, highlighted the importance of the patient voice to improve awareness, and create consistent standard-of-care in Becker. Craig McDonald, Director of the MDA Neuromuscular Diseases Clinics, University of California Davis, USA, explained how understanding of the natural history of Becker can better direct the use of functional measures such as the North Star Ambulatory Assessment (NSAA), as a meaningful outcome in Becker clinical studies. McDonald also outlined clinical advancements with the fast myofibre (Type II) myosin inhibitor, sevasemten (formerly EDG-5506), including new data from CANYON, the largest randomised, controlled clinical trial in Becker to date. CANYON met its primary endpoint demonstrating significant decreases in biomarkers of muscle damage with sevasemten treatment, as well as stabilised ambulatory function (NSAA) trending to improvement, which is a clinically meaningful outcome for patients.

Professor Meissner, what would you consider the 'turning points' that inspired you to specialise in movement disorders, particularly Parkinson's disease?Rascol: When I started as a neurologist and a clinical pharmacologist, there were a lot of interesting developments in movement disorders, particularly Parkinson's disease, based on the understanding of the dopamine hypothesis.That was the main reason for my curiosity in this area.Meissner: For me, it was very simple.It was, in fact, not an active choice, which I never regret.At that time, I wanted to become a neurologist at the Charity Hospital in Berlin, and the only way was to accept a position in the movement disorder group.That was how I found myself on the topic of Parkinson's disease and movement disorders.Q2 In your opinion, what have been the most significant advancements in Parkinson's disease research over the past decade?Meissner: The development of biological tests such as the alphasynuclein real-time quakinginduced conversion (RT-QuIC), which one day might help us make a more accurate diagnosis.Rascol: I would add the importance of physical exercise in disease management and a multidisciplinary approach to the management of patients.Q3 What motivated the initiation of the Lixipark trial?Rascol: There was a background, particularly in logical data, suggesting a link between diabetes mellitus and an increased risk of Parkinson's disease.Neuropathological studies also showed postmortem in the brain of patients with Interview CC BY-NC 4.

Professor Meissner, what would you consider the 'turning points' that inspired you to specialise in movement disorders, particularly Parkinson's disease?Rascol: When I started as a neurologist and a clinical pharmacologist, there were a lot of interesting developments in movement disorders, particularly Parkinson's disease, based on the understanding of the dopamine hypothesis.That was the main reason for my curiosity in this area.Meissner: For me, it was very simple.It was, in fact, not an active choice, which I never regret.At that time, I wanted to become a neurologist at the Charity Hospital in Berlin, and the only way was to accept a position in the movement disorder group.That was how I found myself on the topic of Parkinson's disease and movement disorders.Q2 In your opinion, what have been the most significant advancements in Parkinson's disease research over the past decade?Meissner: The development of biological tests such as the alphasynuclein real-time quakinginduced conversion (RT-QuIC), which one day might help us make a more accurate diagnosis.Rascol: I would add the importance of physical exercise in disease management and a multidisciplinary approach to the management of patients.Q3 What motivated the initiation of the Lixipark trial?Rascol: There was a background, particularly in logical data, suggesting a link between diabetes mellitus and an increased risk of Parkinson's disease.Neuropathological studies also showed postmortem in the brain of patients with Interview CC BY-NC 4.

Lennox–Gastaut syndrome (LGS) is a severe childhood-onset epilepsy syndrome that can be challenging to identify due to its heterogeneous and evolving presentation. During interviews conducted by EMJ in July and August 2023, Karen Keough, a child neurologist specialising in paediatric refractory epilepsy at Texas A&M University School of Medicine, Round Rock, USA, and Rhys Thomas, an adult neurologist specialising in epilepsy (leading on learning disability and genetic testing) at the Royal Victoria Infirmary, Newcastle, UK, and Newcastle University, UK, gave their informed opinions on the challenges of diagnosing LGS. The experts spoke about the characteristic features of LGS, and how its gradual evolution from childhood onset can impede diagnosis. In particular, the issues around diagnosing LGS in adulthood, after considerable disease evolution and against a background of uncertain/incomplete medical history, were discussed. The experts also considered the 2022 International League Against Epilepsy (ILAE) position paper for the definition of epilepsy syndromes in childhood, and its potential impact for directing the diagnosis of LGS. Keough and Thomas concluded by describing how raised awareness, vigilance, and support in diagnosis could help to optimise treatment and readjust the goals for the management of adults and children with LGS.