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The short-term safety and efficacy of IFN-β in patients with multiple sclerosis are well established, but less is known about this drug when taken over years and decades. Sormani and Bruzzi discuss the difficulties associated with designing studies of the long-term treatment effects of IFN-β. They then present techniques that have been employed to minimize potential sources of bias. The authors conclude that long-term use of IFN-β reduces clinical progression, but important questions relating to mortality warrant further investigation.
After brain injuries, microglia and macrophages can aid or hinder tissue repair depending on polarization toward specific cell phenotypes. This Perspectives article describes the phenotypic dynamics and different functions of these cells after acute CNS injury and argues that therapeutic approaches should focus on subtle adjustment of the balance between their phenotypes.
More than 80% of patients with the demyelinating disorder neuromyelitis optica (NMO) experience pain from this condition, which severely affects their quality of life. NMO-associated pain is largely refractory to contemporary pain therapy, suggesting that the mechanisms underlying pain in NMO differ from those underlying other causes of pain. In this article, Bradl and colleagues explore the mechanisms underlying pain in patients with NMO, and attempt to identify molecular and cellular targets for therapy.
Several distinct autoimmune neuropathies can be referred to collectively as Guillain–Barré syndrome (GBS). Wakerley and colleagues argue that confusing terminology hinders diagnosis, and they present a revised classification of 'classic' GBS, Miller Fisher syndrome and related conditions. This new approach to diagnosis might facilitate work-up, thereby helping to identify the correct treatment as quickly as possible.
Preimplantation genetic diagnosis (PGD) facilitates conception of a healthy child by couples at risk of having offspring with an inherited debilitating or fatal neurological disorder. PGD has been carried out for various conditions, including spinal muscular atrophy, Huntington disease, fragile X syndrome, and chromosomal and mitochondrial disorders. This article aims to assist neurologists in counselling and treatment of individuals who wish to explore the option of PGD, and advocates the formulation of guidelines for the responsible integration of PGD into preventative neurology.
Genetic testing has the potential to revolutionize care for individuals with epilepsy, but its advent is bringing to light important new issues, particularly surrounding the benefits and risks of testing, and the optimal provision of genetic services. Poduri and colleagues describe these challenges, and call for mechanisms to be established to address them.
Kullmann et al. discuss the need for gene therapy in epilepsy and the challenges of translating experimental research into the clinic. Furthermore, they outline the potential for gene therapy strategies in pharmacoresistant epilepsy, ways to assess therapeutic efficacy, and the need to evaluate long-term safety data.
