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In this Review of adult diffuse gliomas, Molinaro et al. discuss the latest epidemiological data and highlight advances from the past decade, including the identification of genetic and other risk factors for glioma included in the 2016 WHO integrated classification system.
In this Review, Adams et al. discuss the latest insights into various aspects of hereditary transthyretin amyloidosis and its management, including its epidemiology, pathogenesis and the latest success with RNA interference and antisense oligonucleotide therapies.
Verhaart and Aartsma-Rus discuss important advances in the treatment of Duchenne muscular dystrophy. Gene-addition, exon-skipping, stop codon readthrough and genome-editing approaches aim to restore expression of partially functional dystrophin, whereas treatments that target disrupted pathophysiological pathways can improve muscle function.
Vaughn et al. review the challenges facing elderly patients with multiple sclerosis (MS). They describe the complex, evolving relationship between ageing and MS pathophysiology, highlight the lack of evidence for the safety and efficacy of disease-modifying therapies in elderly patients, and discuss treatment discontinuation and wellness strategies.
In this Review, the authors discuss findings that are transforming our understanding of neuropsychiatric diseases and the role of inflammation in these disorders. They suggest new diagnostic and therapeutic criteria for the emerging field of neuroimmunopsychiatry.
Personalized multiple sclerosis therapy depends on evidence-based prognostication, an initial treatment choice and evaluation of early treatment responses to identify the need to switch therapy. In this Review, Rotstein and Montalban discuss the factors that need to be taken into account to make personalized treatment decisions.
Organic acidurias (OADs) are inherited neurometabolic diseases usually caused by deficiencies in enzymes involved in amino acid catabolism. Wajner reviews the main features of the OADs, focusing particularly on the cerebral manifestations, and highlights recent advances regarding pathophysiology and treatment.
In this Review, Nedelsky and Taylor review the evidence that disturbances in phase transition dynamics and the material properties of ribonucleoprotein granules underlie the pathogenesis of many neurodegenerative diseases, including forms of amyotrophic lateral sclerosis and frontotemporal dementia, among others.
Conventional therapies for Parkinson disease (PD) include dopamine replacement and therapies targeting symptomatic relief, but these approaches fail to modify the underlying disease. This Review explores the novel PD treatment strategies currently being investigated, including pharmaceuticals, cell therapies, immunotherapies, gene therapies and new technologies.
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 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.
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.
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.
