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Rare disease patient groups play a huge role in building a community of people affected by a disease, and helping to share information about research, fundraising, services, clinical care, and symptom management. One of the best ways to help these aims is to run patient group conferences, many of which also give patients the chance to interact with researchers and clinicians in the field. Early in October, Rick spent a day attending not one, but two rare disease patient group conferences to learn more about their role in the rare disease world.

Bright and early one crisp Saturday morning in October, I got in my small silver car and headed to Silverstone. Not, I hasten to add, to partake in a day of motor racing. Rather to attend the morning session of the Wolfram Syndrome UK annual conference. After this I planned to re-route to East Midlands Airport, to join the Ataxia UK annual conference for its closing afternoon, sadly based in the airport itself- rather than somewhere exotic!

Regular blog readers will know that both Wolfram syndrome and Friedreich’s ataxia were selected as example rare diseases for our social impact bond proof of concept study. I had been invited to share the progress of our work with both the Wolfram syndrome and Ataxia communities – unfortunately both conferences fell on the same day! Not one to be daunted by mere logistical difficulties, I decided I wanted to do both. I was very keen to do this as a number of patients and families had been involved in our focus group studies, making a big contribution to the project. I wanted to repay this help by showing how their ideas had helped Findacure, and their own patient organisations.

Importantly for me, these two conferences also presented another opportunity to see a patient conference in action, to meet more people affected by a rare disease, and learn more about the impact of different patient groups on their members.

Wolfram Syndrome UK is a relatively small patient group, focussed on an ultra-rare disease. Despite their size, they have strong ties with the clinical research team at Birmingham Children’s Hospital, as well as the charity WellChild (who co-organised the event). They put on a fantastically professional conference, which I know is highly regarded by Wolfram patients, many of whom attend with their families.

The day started with a focus on research, with talks from Birmingham’s Prof. Barrett, and Washington’s Dr Urano on two different treatment approaches under development for Wolfram syndrome. This included the exciting announcement of funding for a new clinical trial here in the UK. The morning session also included awards for the community’s best fundraisers – a fantastic and personal touch to community building. This was followed by recognition of Wolfram Syndrome UK’s founders, Tracy and Paul Lynch, who were recently awarded a Point’s of Light award by (then) Prime Minister David Cameron. The later stages of the conference included a number of workshops designed exclusively for Wolfram patients, with others for Wolfram parents and carers – an approach designed to allow more freedom of conversation and questioning for both groups. The day also included sports taster sessions (including blind sports), a psychology workshop, and a discussion on building a social life as a Wolfram patient. The blend of science and patient support at the event, was fantastic, and it was a pleasure to be a small part of the day.

At lunchtime, I hopped back into my car in order to attend the Ataxia UK conference, and present our work at the end of the day. Ataxia UK is a large patient organisation, representing a number of different rare ataxias. This translated into many more attendees, but still the same engaged audience, and the same interaction between patient, clinician and researcher. The programme was full, and once again included a mixture of research updates and patient support work. Sessions included a mindfulness workshop, benefits advice, an exploration of the support services provided at a dedicated specialist ataxia centre in Sheffield, a doctor’s Q&A, and a meet the researcher session. The whole event had a friendly, almost celebratory atmosphere. This was particularly obvious at the end of the
day during a fundraising game of “Would I lie to you?” with the Ataxia UK team, and the presentation of the annual awards. I enjoyed the chance to present to one of the largest audiences I’ve ever spoken too, as well as meet some exceptionally interesting people.

Although visiting two conferences in a single day, and driving for four hours in the process, isn’t necessarily the best idea I’ve ever had, it was clearly a fantastic day. Both patient groups, despite their disparity in size, organised professional and informative events, with a real sense of community. It is this latter point – the community spirit engendered by patient organisations – that really struck home for me, and something I am sure that Ataxia UK, Wolfram Syndrome UK, and, with any luck Findacure, will continue to build in the future.


Many thanks to Wolfram Syndrome UK and Ataxia UK for the use of the images provided.

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A patient centric road trip

by Rick Thompson time to read: 3 min