Revista de neurologia
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To define the concepts of orphan drugs and diseases, the current situation of clinical investigation of them, the attitude of the pharmaceutical industry and aspects of health legislation, especially in the European Community. ⋯ The struggle to treat rare diseases must continue, since its true importance is shown when suffered personally.
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Over the past ten years there have been major advances in understanding the neurochemistry of the brain. This has led to better understanding of the mode of action of the classical antiepileptic drugs and the development of new drugs with better pharmacokinetic and pharmacodynamic characteristics. For this reason and also due to their wide mode of action, they have begun to be used for other clinical disorders, particularly neuropathic pain, in the prophylaxis of migraine, in psychiatric disorders and for neuroprotection. ⋯ The new antiepileptic drugs have become an excellent option for the treatment of neuropathic pain. However, for both this and other clinical indications controlled, double blind studies are necessary to determine the efficacy, tolerability and precise indications for these drugs within the therapeutic arsenal available for these disorders.