HSE urged to approve rare disease drug as patients plead for 'more time'
Patients living with Friedreich's Ataxia have urged the HSE to approve funding for a breakthrough treatment, saying access to the drug would give them precious time with their families and slow the progression of the rare neurological condition.
Campaigners gathered outside Leinster House this week ahead of an expected HSE decision on the pricing and reimbursement of Skyclarys, the first treatment approved in Europe for Friedreich's Ataxia.
Friedreich's Ataxia is a progressive neurological disorder that causes irreversible loss of mobility, coordination and speech. While Skyclarys is not a cure, patients say it can slow the progression of the disease, offering valuable extra time and a better quality of life.
The treatment received approval from the European Medicines Agency in February 2024 and is already available in several other European countries. However, it has yet to be approved for reimbursement in Ireland, leaving around 200 patients unable to access the medication.