Emily Felix (28) has remained hopeful and “motivated” that one day a treatment would become available for the rare disease that has shaped her world.
Felix, from Kilkenny, was diagnosed with Friedreich’s ataxia (FA) at the age of 12, one of only 200 people with the condition in the country.
At first, her symptoms included clumsiness, wobbling, struggling to walk and fatigue. It is a rare, inherited and progressive neuromuscular disease.
In February 2023, there was a breakthrough – the omaveloxolone drug Skyclarys was approved in the US and Europe followed in February 2024.
READ MORE
But this long‑awaited therapy remains out of reach in Ireland.
The National Centre for Pharmacoeconomics issued a recommendation regarding the “cost-effectiveness” of Skyclarys that recommended the drug not be considered for reimbursement by the Health Service Executive.
Felix and her family were “killed” by this news which came on her 28th birthday, December 16th, 2025.
She described the recommendation as a “slap in the face”.
“It’s a recommendation, not based on anyone’s views or anyone’s experiences with Friedreich’s [ataxia], but the whole thing is based on cost and money. It’s not nice being reduced to a figure in a budget.
“I have no control over the progression of my disease, but this one thing which could slow down the progression, I believe that it should be my decision,” she added.
Those with FA can also have scoliosis, diabetes or heart conditions such as cardiomyopathy. It can impact every muscle in the body including voice muscles, the swallow reflex, mobility, co-ordination, eyesight, hearing and circulation.
Felix said she has lost sensation below her knees, she has also undergone numerous surgeries for scoliosis and has a heart condition.
She said she does not receive ongoing physiotherapy, occupational therapy and speech and language therapy, instead it is on an “ad hoc” and “reactive” basis.
After secondary school, she studied general business at Dublin City University and completed a master’s degree in human resources from University College Dublin Smurfit School. She now works in a bank and is undertaking her FE-1 exams to become a solicitor.
The disease is progressive. She recalled how she walked into secondary school, used a walker but since starting college, she relies on a wheelchair.
“We’re a forgotten minority and I think it’s very easy when you are a minority to be left to the sidelines ... unless we shout loudly and that’s all we’ve had to do for every single service I’ve ever received in my life.”
She said the drug is not a cure and it won’t reverse the damage that’s already been done, but “will slow it down and it’ll give time and time is what we need”.
[ A quarter of children on Camhs list are waiting more than nine monthsOpens in new window ]
Key decisions “vital to our lives are being made by people in a board room who haven’t been affected by this disease at all”.
Her mother Marie Felix described it as “inhumane”, saying “time is of the essence with a rare disease ... here, there is a treatment and she’s not getting it.”
“The system is so slow, it’s been over 600 days since the application went in to the HSE.”
The family said the drug has the ability to slow the progression of the condition by up to 55 per cent as well as reducing fatigue and improving energy.
“It’s not going to buy back what she has lost. So we need to preserve what she has. This drug is so important now to stop the progression,” said Marie Felix.
In February, Emily and four others with FA attended Leinster House to raise awareness on their need for this drug.
In a statement, the HSE said, it is “committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available [provided] to it”.
Decisions on which medicines are reimbursed are made on “objective, scientific and economic grounds”.
The HSE said it received an application for pricing and reimbursement of Skyclarys on August 1st, 2024 from Biogen Idec (Ireland) Limited.
It said the Drugs Group considers all of the evidence and makes a recommendation to the HSE senior leadership team, the decision-making authority in the HSE.
The HSE also said this application remains under consideration and it cannot make any comment on possible outcomes from the ongoing process.
The Department of Health said: “Ireland’s approach to rare diseases is shaped by its National Rare Disease Strategy, which aims to provide fair, timely, and sustainable access to new and effective treatments for patients with rare conditions.”
It said there was a “notable rise” in waiting times for primary care therapies and to tackle these delays, the department and the HSE have introduced a “targeted programme” to reduce waiting time to under 10 months.
The National Centre for Pharmacoeconomics was contacted for comment.
[ Health service has improved ‘beyond recognition’ in past 20 years, Taoiseach saysOpens in new window ]
