Mother of girl (5) with rare condition hails ‘life-changing therapy’ at Dublin research facility as it marks 10-year anniversary

The CRF supports research and clinical trials across a range of conditions.

Speaking at the event, the facility's director Professor Martina Hennessy called on the Government to invest more in clinical trials, saying Ireland is falling behind the rest of Europe.

"Studies have shown that research-active hospitals are associated with better patient outcomes. In order for us at CRF to continue to treat Ireland's cancer patients and patients with rare diseases it is vital that the facility and clinical research in public hospitals is adequately funded," Prof Hennessy said.

Ireland is falling short of its own National Cancer Strategy target, which aims for 6pc of cancer patients to benefit from clinical trials, she added.

"We are missing this target by 2-3pc currently, and we are falling behind other European countries," Prof Hennessy said.

"The work we are doing in CRF highlights the importance of access to new medicines for cancer patients in Ireland.

"Patients attending the CRF have early access to novel and life-changing therapeutics, for conditions which are often very costly to treat."

Amanda Burke, from Rosmuc in Co Galway, attended the event with her daughter Mollie (5) who was treated for Spinal Muscular Atrophy (SMA) Type 1 at St James's CRF when she was a baby.

CRF is the only clinic in Ireland which offers gene modifying therapy which can slow down the progression of SMA if it is administered early enough.

"We knew Mollie would never be able to walk and would be a wheelchair user, but we wanted her to have as much independence as possible and keep her as strong as possible so she could have a better quality of life," Ms Burke said.

"Mollie underwent the treatment at St James's CRF when she was 1 year and 8 months. It means Mollie hasn't lost all her movement and she can feed herself and walk with the assistance of a walking frame and live a much fuller life thanks to this life-changing therapy."