Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain
the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in
Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles
and JavaScript.
Less than a decade on from genetic studies indicating the potential of PCSK9 as a target to affect cholesterol metabolism, two PCSK9 inhibitors are moving into late-stage development.
Long-standing efforts to develop drugs that target MEKs could soon come to fruition, moving the frontier to how best to combine them with other cancer therapies.
The market for drugs to treat wet age-related macular degeneration (AMD) is expected to more than double by 2016. This article investigates unmet needs and discusses current agents in the pipeline.
Preclinical research indicates that various drugs approved for indications such as hypertension and diabetes could also have potentially beneficial effects in Alzheimer's disease, and for some drugs the evidence is also supported by epidemiological data or preliminary clinical trials. This article presents a formal consensus evaluation of these drug repositioning opportunities, and highlights several compounds for which sufficient evidence is available to encourage further investigation and potential progression to clinical trials in Alzheimer's disease.
A number of compounds, derived from bacterial fermentation products, have been found to target subunits of the spliceosome and display anticancer properties. In this Review, Valcárcel and colleagues discuss the role of splicing in cancer and how insights into the mechanism of action of these bacterial compounds might lead to the development of novel antitumour drugs.
MicroRNAs (miRNAs) are involved in the regulation of gene expression and have been implicated in the pathology of several diseases. Here, van Rooij and Olson discuss the chemistry of current miRNA inhibitors and evaluate miRNAs as potential therapeutic targets for cardiovascular disorders.
Vemurafenib is a small-molecule inhibitor of the oncogenic BRAF kinase approved for the treatment of melanoma. Here, the scientists involved in the discovery of vemurafenib describe the underlying biology of BRAF, the technology used to identify vemurafenib and its clinical development milestones, along with future prospects based on lessons learned during its development.
