Ataxia UK

29/05/2026

On 18th May Design Therapeutics provided an update on their Phase 1/2 RESTORE-FA trial which is taking place in Australia assessing the safety and effectiveness of DT-216P2 in treating FA. The press release provides updates on findings after 4 people took a specific dose of the drug for 4 weeks where they found improvements in clinical measures and dose dependent increases in frataxin mRNA in blood and muscle, with the drug being generally well tolerated. Click this link to for more information: https://www.ataxia.org.uk/research-news/first-fa-patient-dosed-in-restore-fa-trial/

29/05/2026

On 7th May PTC Therapeutics announced that a meeting with FDA (a Type C meeting) was held in April 2026 to discuss a vatiquinone study to support NDA (New Drug Application) resubmission for the Friedreich’s ataxia programme. Based on the meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history controls in Q3 2026. The primary endpoint will be change in mFARS from baseline to 24 months. Read their press release and more about the trial via this link: https://www.ataxia.org.uk/research-news/ptc-therapeutics-announces-that-the-fda-has-not-approved-vatiquinone-to-treat-fa-in-adults-and-children/

28/05/2026

All About Ataxia is an information seminar that gives anyone affected by ataxia (patients, carers/family members) information about the clinical and practical implications of living with ataxia. They explain what is known about ataxia, how it may progress, and what can be done to mitigate its effects

Our next seminar will be taking place on the 31st May 2026 from 10am-2pm.
There will be lots of opportunities to ask questions, and share experiences in small facilitated groups with other participants. There will also be a question and answer session at the event, please could you submit any questions that you have to [email protected]

We look forward to seeing you there!

28/05/2026

Ataxia UK has been featured by Xtalks in a new interview with our Chief Executive, Sue Millman, discussing the urgent need for faster diagnosis, better access to specialist care, and continued progress in ataxia research.

“We want seamless, easily navigable pathways that enable patients to get what they need from our medical services. What people with ataxia want, in common with all the rest of the various medical conditions, is a speedy diagnosis.”……

Read more here.

Gain insight into the urgent need for faster ataxia diagnosis, improved access to emerging therapies and stronger support for patients and families.

27/05/2026

Join us this Carers Week (8th-14th June) for a week of FREE virtual activities as we celebrate and give thanks to our carers for their contributions to those in the ataxia community.

Coming up in Carers Week:
- Tuesday 9th June, 1-2pm – Mindfulness
- Wednesday 10th June, 2-3:30pm - Carers Support Group
- Friday 12th June, 12:30-2:30pm - All About Ataxia Plus: Care Needs

If you are a carer looking for more information on the support available to you, please have a look at the Carers UK website or contact our Helpline at [email protected].

Check out our website for the booking form and to find out more.

26/05/2026

Don't miss your chance to join us at the Liverpool Roadshow!
The last few tickets are available - book yours before booking closes at midnight on the 29th May.
This in-person event is a great opportunity to find out more about ataxia and the support that Ataxia UK can offer. You'll be able to hear the latest research updates, put your questions to a specialist ataxia neurologist and meet others living with ataxia.

https://www.ataxia.org.uk/support-services/ataxia-uk-conferences/liverpool-roadshow/

26/05/2026

Volunteers week
Join us next week for Volunteers Week (1st-7th June) as we celebrate our volunteers and their great contribution to the ataxia community! In this week, we will hosting a range of FREE Virtual Activities.

Coming up in Volunteers Week:
- Tuesday 2nd June, 11am-12pm - Chair Pilates
- Thursday 4th June, 11am-12:30 - AbilityNet: The Basics

Check out our website for the booking form and to find out more.

https://www.ataxia.org.uk/volunteers-week-2026/

23/05/2026

A huge thank you to Scarlett Parr-Reid for delivering the latest research updates on cerebellar ataxia and Friedreich’s ataxia at our Glasgow Roadshow 2026.

Keeping our community informed about developments in research is such an important part of these events, and we’re grateful for Scarlett’s expertise and insight. 🔬💙

Our next Roadshow takes place in Liverpool on 7 June — don’t miss it!
Bookings close on Friday 29 May
https://www.ataxia.org.uk/support-services/ataxia-uk-conferences/liverpool-roadshow/

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23/05/2026

Congratulations to Kelly, Prof Paola Giunti's (Lead of the London Ataxia Centre) secretary, who has won the UCLH administrator award in recognition of outstanding patient experience!