In the latest edition of touchREVIEWS in Neurology, we are pleased to present a collection of insightful articles that highlight the current landscape and future directions in neurological research and treatment.
Firstly, Rajvinder Karda opens this issue with a compelling expert interview on the future of SCN1A gene-targeting research for the treatment of Dravet syndrome. Dr Karda’s insights offer a glimpse into the current status of SCN1A gene-targeting research, and innovative approaches being explored to tackle this challenging genetic disorder.
Following this, Ralf Reilmann discusses the pivotal PROOF-HD phase 3 results investigating pridopidine in Huntington’s Disease. This interview highlights the trial outcomes, drawing comparisons to current treatment options. The interview also addresses why the primary endpoint was missed and outlines potential avenues for future research and development.
In an informative editorial, Nancy L. Kuntz and Bridget McGowan provide an update on vamorolone and steroids in the treatment of Duchenne muscular dystrophy. The article offers an understanding of vamorolone, a novel, first-in-class, dissociative steroid, and its implications for muscular dystrophy management, and a comparison to classic corticosteroid use.
Next, Riley Kessler, Sonal Sharma and David Lynch contribute a comprehensive review on omaveloxolone for the treatment of Friedreich’s ataxia. This article thoroughly examines omaveloxolone’s role in addressing a rare but debilitating neurodegenerative condition, highlights the major clinical trials leading to its approval in the USA, and a discussion of the challenges faced in clinical trials. The review also provides a perspective on other therapies under investigation.
The issue also includes two intriguing case reports. Prashant Bhatele and Manoj Khanal investigate a rare presentation of steroid responsive encephalopathy associated with autoimmune thyroiditis with neuropsychiatric symptoms.
Second, Cathal Ahern’s case report on the paradoxical tumefactive worsening of multiple sclerosis (MS) following initiation of natalizumab. This report offers a critical look at a rare but serious complication of MS treatment, contributing to a deeper understanding of MS management complexities.
The editors of touchREVIEWS in Neurology would like to thank everyone who contributed to the insightful and informative articles included in this edition. We are also grateful to all society partners for their on-going support and to our Editors-in-Chief and Editorial Board for their continued involvement and advice. We hope that you will find this edition informative and interesting.
Cris S Constantinescu
Prof. Cris S Constantinescu is the co-Editor-in-Chief of touchREVIEWS in Neurology. Cris Constantinescu is Attending Neurologist at Cooper University Hospital, Cooper Neurological Institute, Cherry Hill, NJ, USA, Professor of Neurology at Cooper Medical School of Rowan University, Camden, NJ, USA, and Emeritus Professor of Neurology in the Academic Unit of Mental Health and Clinical Neurosciences at the University of Nottingham, Nottingham, UK. He graduated with an MD from Boston University School of Medicine, USA, in 1988. As a recipient of the physician-scientist award from the National Institutes of Health, USA, he completed a PhD in immunology at the University of Pennsylvania in 1998. He has completed an internship and a residency in neurology and fellowships in neuroimmunology and neurorehabilitation. He was appointed honorary consultant neurologist and senior lecturer in neurology at the University of Nottingham 2000–2001. In 2004, he was appointed Professor of Neurology and Chair in Neurology at the University of Nottingham, a post he held until 2021. He has authored or co-authored over 200 articles in international journals. His research interests are immune regulation in inflammatory diseases of the nervous system, in particular multiple sclerosis, neuroimaging, clinical trials and cognitive neurology. He is currently involved in the care of numerous patients with neuroimmunological and neurodegenerative diseases.
