Mar-16 – Ataxia



Ataxia UK got excellent coverage for the Rare Disease Day

Our research officer Dr Julie Vallortigara was invited to speak at the Findacure event for Rare Disease Day to present patient group led initiatives on drugs repurposing to find treatments for the ataxias. You can listen to the talk and see slides here. Dr Vallortigara also got the opportunity to give an interview for Disease Spotlight, to talk about rare disease and raise awareness about ataxia. You can read this interview here.

Posted: 11/03/2016

EMA launched a new program for faster drugs approval

The European Medicines Agency (EMA) launched a new initiative Monday called PRIME, which is designed to help expedite the regulatory process for drug makers working on promising medications for rare diseases. You can read the program’s guidelines on the EMA website here.

Posted: 10/03/2016

Retrotope advances Friedreich’s ataxia trial

Following announcement of the opening of a Friedreich’s ataxia trial at the International Ataxia Research conference in March 2015, it is encouraging to see that the first cohort of patients have now completed the trial with no major side effects. The next group of participants are being recruited to this US trial now. More information in the Ataxia magazine issue 190.

Posted: 08/03/2016

Genome editing explained

Genome editing has been on the news recently due to the progress being made in these scientific techniques. Genetic Alliance UK has produced an interesting paper on this topic. You can take a look at their in-depth summary.

Posted: 01/03/2016


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