Ataxia UK 23/24 review

The ataxias are a set of life-limiting neurological conditions that disrupt the messages sent from our brains to our muscles. Not just the muscles we use to move, but those we use to speak, listen and see. Over time, people with ataxia are slowly imprisoned in their own bodies, losing the tools they once had to communicate with loved ones. Over 200 different genetic ataxias have been discovered in the past 30 years, each of which requires a different treatment. Currently, no treatments or cures for most of the ataxias have been found. Ataxia UK is the leading national charity which supports those affected ataxia and funds research into finding treatments and cures.

The most common and aggressive form of the condition is Friedreich’s ataxia (FA), which predominantly shows its symptoms during childhood or early puberty. The earlier FA begins to show, the likelihood is the faster it progresses. It hampers a child/young person’s balance, mobility and movement, before causing speech, hearing and vision to deteriorate and, eventually, weakens the heart. As a result, children diagnosed with FA usually have a shorter life expectancy than their peers and will spend much of their life in a wheelchair.

As with the other ataxias, there is no cure for FA.

A major part of our work is to support and advise parents of children who have FA, whilst funding research into treatments and a cure. The support we receive from Childlife is essential in achieving this: it enables us to run local support groups, conferences, various online forums and information sessions to reduce families’ isolation and help them understand the condition.

Some key highlights from FY 2023-24

After a successful International congress for ataxia research in late 2022, Ataxia UK is again partnering with two other ataxia charities to organise and host the next congress in November 20242 in London. This is the ‘go-to’ ataxia conference for researchers worldwide, where we anticipate around 500 delegates will attend, share the latest research developments and importantly will network and set up new collaborations to advance ataxia research.

Just over a year ago we were delighted as we had the first drug approved for the treatment of Friedreich’s ataxia. For now the approval is only for people aged 16 years and over and for people living in the US (approval Feb 2023) and EU countries (approval 2024).

Ataxia UK has been working hard to encourage and support Biogen (who have the license for Skyclarys) to seek approval in the UK and we are hopeful this will happen soon. There are also plans for a trial testing the drug in children to start this summer, so that we can hopefully get to the point of having an approved drug for those over 16 years old too.

Ataxia UK is following other exciting developments with trials starting testing gene therapy in people with FA, and a therapy targeting genes affected in people with SCA1 and SCA3 starting soon at the Ataxia UK accredited Ataxia centre in London.

In addition to these centres, the Ataxia UK Helpline continues to be the cornerstone of providing assistance and guide those affected by ataxia. In 2023-24 the Helpline received a total of 1350 contacts to the Helpline, which supported 996 individuals with 1645 issues including:

• Information about ataxia, health needs, therapies, referrals and diagnosis.
• Social care needs inc. housing, aids and adaptations.
• Welfare benefits, grants and finances.

The Helpline also welcomed 4 new volunteers to Helpline but said goodbye to 2 volunteers.

To make Ataxia UK’s services available to as many people in the community as possible, we’ve also held the first-ever hybrid conference in late October 2023 in East Midlands. This event saw 120 people attend in person, with some hundred more joining the live stream across the weekend. Building on the success of this event, Ataxia UK will also be delivering a travelling Roadshow in Cardiff, Edinburgh & Belfast to bring the services and information most needed to those affected by ataxia.

Viktor is the Senior Brand and Communications Manager at Childlife member charity, Ataxia UK