Articles in 2020

Current medications used for pain management can have dose-limiting adverse effects. In this Review, Waxman et al. discuss compounds designed to target peripheral sodium channels for pain relief and highlight the challenges and future potential of this therapeutic strategy.

Focused ultrasound (FUS) offers the ability to non-invasively and precisely intervene in key circuits that underlie common and challenging brain disorders. This Review provides a comprehensive update of FUS applications in the brain, including thermoablation, blood–brain barrier opening and neuromodulation.

The anti-seizure medications used to treat patients with epilepsy can improve symptoms but do not address the underlying cause of the condition. In this Review, the authors discuss the ongoing shift towards personalized treatments for specific epilepsy aetiologies.

In this Review, the authors discuss how new therapies are changing the field of spinal muscular atrophy. They consider the effects of treatment at different stages of the disease, what treatment effects tell us about the disease and the challenges facing the field in the treatment era.

Despite the development of many new anti-seizure drugs over the past two decades, around one-third of individuals with epilepsy are without effective treatment. This pharmacoresistance is poorly understood, but new treatments targeting epileptogenesis instead of seizures have shown potential in animal models and are now being translated into the clinic.

Following extensive progress in the treatment of relapsing multiple sclerosis, the major challenge in the field is now to develop effective therapies for progressive forms of multiple sclerosis. As the first signs of success emerge, now is the time to consider the research needed to move the field forward.

Advances in neurology over the past 15 years have transformed the treatment of a number of conditions but have also raised new questions and challenges.

In this Review, Dalakas et al. discuss the complement system, the role it plays in autoimmune neurological disease and neurodegenerative disease, and provide an overview of the latest therapeutics that target complement and that can be used for or have potential in neurological disorders.

The introduction of therapies for spinal muscular atrophy (SMA) has rapidly changed the clinical landscape, transforming SMA from a lethal to a treatable disease. This transformation has driven further advances, from population screening imperatives to novel treatment delivery approaches, while uncovering health disparities and fuelling debate regarding drug pricing.

Despite the strong treatment effects of mechanical thrombectomy in acute ischaemic stroke, penumbral tissue loss before recanalization and ischaemia–reperfusion injury after diminish functional outcomes and call for adjunct treatments. Classical neuroprotection strategies could consequently be revived, but novel treatment targets are also emerging through mechanistic research.

Despite negative findings from numerous clinical trials of potential disease-modifying therapies for Alzheimer disease, amyloid remains the most compelling therapeutic target. Advances in biomarker methods now enable accurate monitoring of Alzheimer disease progression from the earliest stages of the disease. We must therefore redouble efforts to find an effective treatment.

Drug repositioning and repurposing can enhance traditional drug development efforts and could accelerate the identification of new treatments. In this Review, Ballard and colleagues highlight priority compounds for repurposing for the treatment of Alzheimer disease.

B cell-depleting agents are emerging as important disease-modifying drugs for multiple sclerosis, but their effectiveness in relation to established treatments remains uncertain. To cast light on this issue, several studies have provided head-to-head comparisons of the anti-CD20 antibody rituximab with natalizumab, fingolimod and dimethyl fumarate in patients with multiple sclerosis.

With the advent of disease-modifying therapies for spinal muscular atrophy, prenatal and extra-neural alterations associated with the condition have garnered increasing attention as potential determinants of the therapeutic window and efficacy of novel drugs. Two recent studies highlight the impact of spinal muscular atrophy on prenatal bone and organ development.

Malformations of cortical development (MCDs) are neurodevelopmental disorders that result from abnormal development of the cerebral cortex in utero. In this Consensus Statement, the international MCD network Neuro-MIG provides recommendations to aid both expert and non-expert clinicians in the diagnostic work-up of MCDs.