'Time is muscle' - decision urged on rare disease medicine

The parents of Aaron Langan, a nine-year-old boy with Duchenne Muscular Dystrophy (DMD), are calling on Ireland’s government to accelerate access to Givinostat, a drug approved by the European Medicines Agency in June 2025. The drug slows disease progression but has not been approved for reimbursement in Ireland, leaving fewer than 200 affected individuals without treatment options. A public assembly in Donegal town was organized to demand immediate action, with parents Sinead and Dermot Langan emphasizing that delays mean irreversible loss of muscle function, using the phrase ‘time is muscle.’ Aaron, who currently uses a wheelchair and requires daily care, is one of several children affected by DMD in Ireland. His parents, alongside other families, have repeatedly pressed the government and the Health Service Executive (HSE) to fast-track the drug’s approval. They argue that the UK’s definition of ‘ambulatory’—used to determine eligibility—should be adopted in Ireland to ensure children like Aaron can access the treatment sooner. The Department of Health confirmed Minister Jennifer Carroll MacNeill remains engaged with families and is monitoring the reimbursement process. The HSE received a commercial proposal from Italfarmaco, the drug’s manufacturer, on May 8, 2026, but negotiations on pricing and cost-effectiveness are still underway. The HSE Drugs Group will review submissions from patient groups, economic reports, and negotiation outcomes before making a recommendation to senior leadership. Parents expressed frustration over the year-long delay, stating their children deserve better and warning further postponement would be ‘devastating.’ The issue has also been raised in Ireland’s parliament, with lawmakers urging the government to align with the UK’s access model. As negotiations continue, families remain hopeful but insist urgent action is needed to prevent further irreversible damage for children with DMD.