“Begging for access to medicine" - TDs highlight Mayo families rare disease challenges in Dáil
Mayo TDs call for faster access to life-changing treatments during Dáil debate
Families affected by rare diseases in Mayo were brought into sharp focus during a Dáil debate on rare diseases yesterday (Thursday, June 18), with local representatives urging the Government to speed up access to potentially life-changing medicines and improve supports for patients facing progressive conditions.
During the debate, Mayo TDs highlighted the experiences of people living with rare diseases, including Ballyhaunis man Eric Fitzgerald, who is living with Friedreich’s ataxia, a rare inherited neurological condition that causes progressive damage to the nervous system and loss of muscle function.
Speaking in the Dáil, Deputy Paul Lawless said Eric’s condition continues to worsen as time passes.
“For Eric, the passage of time means the loss of muscle. His condition is getting progressively worse. It is extraordinarily sad,” Deputy Lawless said. “I met a group of young people with Friedreich's ataxia last month.”
“I met a wonderful young woman called Emily Felix. Emily has started recording her voice because she is losing her voice and losing muscle. That tells you how painful this disease is.”
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The Mayo TD also referred to other patients living with Friedreich’s ataxia, describing the impact the condition has on families as they await decisions on access to new treatments.
A key focus of the debate was Skyclarys, a medication approved by the European Medicines Agency in 2024. While not a cure, clinical studies suggest the treatment can slow the progression of Friedreich’s ataxia, offering hope to patients and families.
“There is some hope, however, and that hope is in the form of the medication Skyclarys. It was approved by the European Medicines Agency in 2024,” Deputy Lawless said. “While it is not a full cure, the clinical guidelines show that it will reduce the progression of the disease by half for some patients.”
Deputy Rose Conway-Walsh told the Dáil that families in Mayo and across Ireland are anxiously awaiting a decision on the drug’s reimbursement through the HSE.
“I want to raise the urgent need for access to Skyclarys. It has been raised many times in the Chamber,” she said. “There are over 200 people living with Friedrich's ataxia in Ireland. For those diagnosed with this devastating rare condition, this drug represents far more than another medicine. It offers hope for slowing progression, preserving mobility and maintaining independence and giving families precious time together.”
“Every month that passes means a loss of function that can never be regained,” she added. “Time matters enormously for people with progressive degenerative conditions.”
She noted that Skyclarys is already available in several countries, including the UK, France, Germany and Austria, while patients in Ireland continue to wait for a funding decision.
The debate comes as the Government advances the National Rare Disease Strategy 2025-2030, which aims to improve diagnosis, treatment and support services for the estimated 300,000 people living with rare diseases in Ireland.
Minister for Health Jennifer Carroll MacNeill said the strategy is focused on earlier diagnosis, better coordinated care and improved access to treatments. She acknowledged the difficulties faced by families navigating complex healthcare systems and lengthy diagnostic journeys.
On the issue of rare disease medicines, she confirmed that the Government is examining new pathways for earlier access to orphan drugs and is carrying out a comprehensive review of Ireland’s medicines approval and reimbursement system.
While no decision has yet been made on the reimbursement of Skyclarys, the Minister said the application remains under active consideration by the HSE.
The debate also heard positive news for families affected by Duchenne muscular dystrophy, with confirmation that the drug givinostat has been approved for reimbursement following a recommendation by the HSE’s drugs group.
For Mayo families affected by rare diseases, however, representatives stressed that faster access to treatments remains a critical issue.