Prader-Willi syndrome. Fabry disease of the kidney. Spinocerebellar ataxia. Only a few people have heard these and the other ' rare diseases ', only hundreds of patients worldwide influence of some. " On the search for the next blockbuster pharmaceutical companies pay them little attention. But the diseases are generally incurable - and there are thousands of them.
Make it more difficult this week, the US national institutes of health (NIH) and the European Commission launch a joint attack on these conditions to test the small number of new therapies and Diagnostics to develop. The international rare disease research consortium under the auspices of the two bodies formed the ambition has a diagnostic tool develop for each known rare disease by 2020, along with new therapies to treat 200 of them. "The number of persons that one particular rare disease is so small that we need to pool information from patients in as many countries as possible," says Ruxandra Draghia-Akli, the Commission Director of health research.
"We need to can pool information from patients in as many countries as possible."
At the start meeting in Bethesda, Maryland on 6 - 8 April, be associated with potential partners from research strategies identify diagnostic biomarkers to design clinical trials and to coordinate the sequencing of the genome in these diseases. Almost all rare diseases, of which there are an estimated 6, 000-8, 000, are the result of small genetic changes.
The meeting will be modelled also the governance of the project, discussed most likely on the groundbreaking human genome project. As such, the Consortium for research agencies and organizations from around the world is open. Representatives from countries such as Canada, Japan, and some European Nations are all in the meeting and can join the Consortium. Those who have to attend promise a minimum financial contribution still not agreed, and all relevant data to share. The project must indeed, exchange of information, such as the fact that doctors in different countries use the same disease often entirely different words, describe numerous obstacles to overcome.
Draghia Akli points out that the project great benefits to the emerging field of personalized medicine could - bring another political priority for the NIH and the Commission - which also is the challenge of small populations of patients.
Regulatory agencies as that left US Food and drug administration and the European Medicines Agency on large, randomized and controlled clinical trials to decide whether new drugs to approve, and one of the objectives of the Consortium, to develop clinical trials of alternative methods for diseases, which will affect only a few people.
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These methods are more and more important now that genome analysis helps reduce common diseases in smaller and smaller subclasses. "Soon there will be no disease called breast cancer," says Draghia Akli. Instead, the term by "a variety of rare diseases" says replaced harm growth in the breast tissue and individual treatment required each caused it.
The Commission is launching a 100 - million € (US$ 140 - million) Research tender in July, which scientific objectives by focusing on the development of appropriate clinical trials will strongly support the Consortium.
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