Progress in brain research
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glaucoma is a remarkable social issue being the leading cause of irreversible blindness worldwide. It is a progressive neuropathy characterized by the death of the retinal ganglion cells, of which the most important risk factor is represented by the increase of intraocular pressure (IOP). The role of nutraceutical supplementations with anti-oxidant activity has been extensively tested in preclinical models of glaucoma. The clinical efficacy of nutraceuticals in glaucoma is still controversial. ⋯ the evidence of effectiveness of nutraceutical formulations is still uncertain and inconclusive. Therefore, large double-blind randomized clinical trials with adequate design, methodology and statistical power are needed to support the use of nutraceuticals in glaucoma.
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Migraine is a highly heritable complex brain disorder, imposing a huge burden of disability on sufferers. The genetic architecture of migraine ranges from the rare Mendelian forms whereby a single gene mutation is sufficient to cause disease to gene variants that individually impart only a small increase in migraine risk. Despite the considerable advances in the last decade, there are significant challenges to translate genetic findings into drug targets and eventually successful treatments. ⋯ This will require integration of genetic data with new technologies such as human stem cell models of migraine that allow the interpretation of genetic risk into disease relevant cellular phenotypes. This was recently undertaken for the first time in migraine, whereby stem cells from patients with the rare TRESK frameshift mutation converted into pain sensory neurons demonstrated hyper-excitability. The continued study of the molecular basis of migraine thus offers new paths to drug targets and precision medicine approaches.
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Review
A new era for migraine: The role of calcitonin gene-related peptide in the trigeminovascular system.
There is a huge improvement in our understanding of migraine pathophysiology in the past decades. The activation of the trigeminovascular system has been proved to play a key role in migraine. Calcitonin gene-related peptide (CGRP) and CGRP receptors are widely distributed in the trigeminovascular system. ⋯ Based on these findings, several treatment options have been designed for migraine treatment, including CGRP receptor antagonists (gepants) and monoclonal antibodies targeting CGRP or CGRP receptors. The clinical trials show both gepants and monoclonal antibodies are effective for migraine treatment. In this section, we describe the roles of the trigeminovascular system in migraine, the discovery of CGRP, and the CGRP signaling pathway.