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    Many of the thousands of rare diseases lack effective treatments. However, economic and regulatory incentives, as well as advances in our understanding of disease aetiology, have encouraged increased investment in the development of drugs for rare diseases — orphan drugs. This special collection from Nature Reviews Drug Discoveryincludes articles that discuss the impact of orphan drug legislation in the United States and Europe, and that review recent therapeutic progress for two groups of rare diseases: neuromuscular disorders and myeloproliferative neoplasias.

  • Collection |

    The rapid growth in the prevalence of type 2 diabetes is increasing the need for effective therapeutic interventions. However, although multiple classes of diabetes drugs are available, achieving long-term control of blood sugar is often a major challenge, in part owing to treatment-limiting adverse effects. With these challenges in mind, this special collection of articles from Nature Reviews Drug Discoveryfocuses on emerging therapeutic approaches and targets for type 2 diabetes.

  • Focus |

    To highlight the problem of resistance to antimicrobials, Nature Reviews Microbiology presents a set of specially commissioned articles that focus on key issues in the field, including the role of antibiotic resistance in nature, how the spread of antimicrobial resistance could be limited and the pharmaceutical strategies that are used to overcome resistance to various classes of antimicrobials. The Focus issue is accompanied by a joint Web Focus with Nature Reviews Drug Discovery.

  • Focus |

    Apoptosis, the major form of controlled cell death, has a key role in the pathogenesis of diseases including cancer, inflammation and neurodegenerative disorders. The past decade has witnessed tremendous progress in understanding of the molecular mechanisms of apoptosis regulation, which has revealed a number of highly promising therapeutic targets, particularly for anticancer strategies. This focus provides a cutting-edge overview of the science and business of targeting apoptosis.

  • Focus |

    Unrelieved pain associated with conditions such as arthritis, diabetes, cancer and serious injury represents a major unmet medical need. With the aim of highlighting approaches to tackling the challenges in this field, this special collection integrates articles that discuss strategies to drive forward analgesic drug development with those that explore new targets for pain therapeutics.

  • Focus |

    This issue provides several articles focusing on antiviral therapy. Additional material, brought together in a joint focus with Nature Reviews Drug Discoverysupported by our principal sponsor Pfizer and our supporting sponsor Gilead.

  • Focus |

    A collection of articles from Nature Biotechnology, brought together in a joint focus with Nature Reviews Drug Discovery, on the science and business of antibacterial discovery and development.

  • Focus |

    For the past decade, the number of molecular targets for approved drugs has been debated. However, to develop and apply predictive methods to assess the future potential for therapeutic exploitation, the number, characteristics and biological diversity of these targets needs to be established. Here, we provide a comprehensive survey of current drug targets and drugs that modulate them.

  • Series |

    A special series of articles discussing challenges and opportunities in the discovery, validation and application of biomarkers.

  • Series |

    A special series of articles discussing the potential uses and limitations of existing and emerging model organisms in drug discovery.

  • Focus |

    We examine the role of kinase signalling in cancer and the potential for developing effective therapies that can modulate various kinase pathways. Following the success of targeted kinase inhibitor therapies such as imatinib and gefitinib this continues to be a fast moving area of research.

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