Workshop: Help to plan a future clinical trial

We are working closely with Dr Wilkins at the University of Bristol in planning a clinical trial in Friedreich’s ataxia (FA), testing a medication called G-CSF. This will be a larger trial, following on from a successful pilot trial – the results of which we hope to publish in the coming months. As part of the trial planning, we think it is really important to capture the opinion of those that will be most affected by a new treatment – people with FA and their families.

We would like to invite those who are interested in discussing the design of the trial to attend a meeting at the Ataxia UK offices in London on Tuesday 15 October. Numbers are limited, so places will be given on a first come, first served basis. If you are interested in attending, or would like more information, please call the office or email [email protected].

Research survey: Patient attitudes to clinical trials in ataxia 

The Research Team at the London Ataxia Centre (University College London Hospitals NHS Foundation Trust and Queen's square Institute of Neurology) has developed a survey in collaboration with Ataxia UK and FARA to investigate the attitude of patients towards clinical trials in ataxia in the UK. This survey is important for helping us to design better trials for the future with patients in mind. Anyone with ataxia is invited to take part, the survey may also be completed on behalf of someone with ataxia by a carer, partner or parent. If you are interested in taking part please read this patient information sheet and then the survey can be found here.


Research survey: Patient attitudes to genome editing for prevention of heritable disorders: Social and Ethical implications

You have been invited to take part in a research study conducted by University College London (UCL). The study aims to explore the opinions and knowledge of those in heritable disease communities regarding genome editing and its applications.

This research is in the form of an online survey. The survey will explore the different aspects of the technology itself, as well as its applications in a medical setting for the treatment of heritable disease. The study will also explore the attitudes of people with experience of heritable diseases towards the introduction of genome editing in embryos for correction of mutations.

We are looking for a variety of people within these communities, those affected and also their family members. Additionally, anyone who is involved in the community but does not have a family member affected is also welcome to join.

You can find the survey here. The survey can be completed via the weblink on smart phones, on laptop or desktop and should not take more than 15 minutes to complete. The deadline to complete this survey is 30 May 2019. We really hope to broaden the discussion to allow all disease communities to be heard. Your time and input are truly appreciated.