What is gene therapy?

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Around 80% of rare diseases have a genetic cause. Many of these diseases have no good treatment options, but gene therapy might help. So what is gene therapy, and how does it work? Raremark have produced an article we think … Continued

Let’s push Genida forward

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We have had a message from GenIDA asking for our help in getting the number of participants in the GenIDA database up from 60 to over 100. This is perfectly possible if just a few more of you could complete … Continued

The good work of ERN-ITHACA

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Members of our medical advisory group work in collaboration with The European Reference Network on Congenital Malformations and Rare Intellectual Disability – thankfully known as ERN-ITHACA to progress research. Just for your information the organisation has been supporting research in … Continued