Time is Muscle: MDI Calls for Transparent Timelines on Treatment Access
MDI CEO Alan Breathnach and Clinic Liaison Officer Sinéad O'Brien were in Leinster House this morning. MDI were supporting campaigning families "Time is Muscle" and had the opportunity to meet with Minister for Health and presented to a full AV room of TD's and Senators alongside our MDI Families campaigning for access to Givinostat.
Families received encouraging news this week following a positive recommendation for Givinostat from the HSE Drugs Review Group. While this represents an important milestone for people living with Duchenne muscular dystrophy, several further steps remain before patients can access treatment through Irish clinics.
Alan Breathnach highlighted that three important therapies — Givinostat for Duchenne muscular dystrophy, Risdiplam for spinal muscular atrophy (SMA), and Skyclarys for Friedreich's ataxia — remain unavailable to many MDI members. Although each treatment is at a different stage of the reimbursement process, patients and families continue to face significant delays.
MDI called on TDs and Senators to seek a transparent timeline and regular progress updates from the Department of Health, the HSE, and the NCPE on all rare disease and neuromuscular drug reimbursement applications, including Givinostat, Risdiplam, and Skyclarys.
Patients and families understand that not every reimbursement decision will be positive. However, they are entitled to a process that is timely, transparent, equitable, and accountable. If Ireland has committed to a rapid review pathway for rare disease treatments, patients deserve clear evidence that the system is delivering on that commitment.