Reviews & Analysis

In the past few years, substantial progress has been made towards effective treatment of Batten disease, a family of paediatric neurodegenerative lysosomal storage disorders. In this Review, the authors discuss new therapies for Batten disease, including cerliponase alfa, the first globally approved agent, and considers promising new therapeutic avenues for future treatments.

In this Review, Limousin and Foltynie discuss the current evidence on the long-term outcomes of deep brain stimulation in Parkinson disease and consider the clinical implications of these findings for future use of this therapeutic approach.

Alzheimer disease (AD) pathology is found in almost all adults with Down syndrome (DS), primarily owing to overexpression of APP, present on chromosome 21. Here, Lott and Head examine the commonalities and disparities between DS and AD and highlight findings in DS that can inform research into AD in the general population.

In the past few years, a number of new migraine treatments have emerged, including monoclonal antibodies against calcitonin gene-related peptide (CGRP) and the CGRP receptor. Now, a position statement from the American Headache Society offers guidance on the integration of these agents into clinical practice in the prevention of migraine.

In this Review, the authors discuss the evidence that exosomes could be used as a neurorestorative therapy after stroke or traumatic brain injury and how engineering of their microRNA cargo could optimize this approach.

Abnormalities in sensory functions, including tactile, proprioceptive and nociceptive processing, are increasingly recognized in patients with focal dystonias. The authors explore the link between sensory system abnormalities and dystonic motor activity in the context of the 'network model' of dystonia.

A new analysis has found that concussion and mild traumatic brain injury (mTBI) are linked to an increased risk of suicidal behaviours and thoughts. However, a host of risk factors might influence this correlation, and careful investigation is required to establish which individuals with mTBI might be most at risk of suicide.

Over the past 20 years, deep brain stimulation (DBS) has transformed the treatment of movement disorders. Now, new therapeutic possibilities for DBS are emerging for other neurological and psychiatric disorders. This Review considers the clinical and scientific advances facilitated by DBS and the crucial questions, challenges and opportunities that face this technology.

In this Perspectives article, Smith et al. address the misconceptions about the clinical and pathological features of chronic traumatic encephalopathy that cause confusion and controversy not only in the public but also among health-care professionals.

In the past few years the scientific community has witnessed a prodigious surge in research activity, publication of data and progress in understanding the mechanistic components of migraine. This renaissance is the result of efforts initiated decades ago that are finally being translated into benefits for individuals affected by this disease.

In 2018, developments in Parkinson disease (PD) research yielded improved diagnostic criteria and provided evidence for the effects of some treatments, both old and new. These developments enrich the treatment options available for PD and are likely to change important guideline recommendations.

The past year saw progress in acute treatment of ischaemic stroke, but large inequalities in stroke services were revealed, warranting strategical initiatives to improve treatment access. Reclassification of stroke as a disease of the nervous system in the WHO International Classification of Diseases 11th revision is likely to help such initiatives.

2018 saw the failure of several large clinical trials that were based on the premise that reduction of amyloid-β levels is an effective treatment for symptomatic Alzheimer disease. Yet, over the same time period, good news also emerged about the diagnostic value of tau PET imaging.

Publications on epilepsy in 2018 have shed light on the aetiology and management of the condition and raised new questions. Translation from mechanisms to clinical practice, driven by cooperation among multiple fields, will be crucial to further advances.

Chronic migraine is a debilitating condition that arises from the worsening of episodic migraine. A large population-based questionnaire study showed that individuals with chronic migraine reported more sleep apnoea and worse sleep quality than individuals with episodic migraine. This finding provides important insights into the association between sleep disturbances and chronic migraine.

In 2018, the distinguishing pathological features of white matter lesions in patients with progressive multiple sclerosis (MS) were refined, and serological and MRI biomarkers of clinical worsening and evolution to progressive MS were identified. We also saw therapeutic advances in progressive MS with the emergence of new neuroprotective strategies and putative markers of neurodegeneration.

Potential disease-modifying therapies for Alzheimer disease have mostly targeted brain accumulation of amyloid-β, but this approach has yet to provide substantial clinical benefits. The authors consider the reasons for this failure and suggest alternative strategies, including modification of risk factors.