Give Irish Duchenne Boys Urgent Access to Givinostat.

Our sons live with Duchenne Muscular Dystrophy (DMD) — a rare, progressive condition that slowly steals their strength, independence, and, too often, their futures.

But now, for the first time, we have something to slow it down: Givinostat — a treatment that can preserve muscle, extend mobility, and protect dignity.

It’s already available in parts of the EU, and available in the UK and US but here in Ireland, our boys are still waiting.

Right now, the HTA(Health Technology Assessment) of Givinostat by the NCPE(National Centre for Pharmacoeconomics) has been completed.

The NCPE has recommended that the treatment should not be reimbursed unless cost-effectiveness can be improved, which means negotiations are now expected to begin.

This is not the final decision.

Following negotiations, the Health Service Executive will consider the medicine for reimbursement, taking into account broader criteria under the Health (Pricing and Supply of Medical Goods) Act 2013 — including patient impact, unmet medical need, and social and ethical considerations, not cost alone.

This means we still have an important window to show decision-makers that there is strong public and political support for getting this treatment to Irish boys as quickly as possible.

Please keep signing and sharing - because time is muscle.

Every delay costs more than time - it costs strength, eligibility, and hope.

  
🙏 What We’re Asking For

1. Speed Up the Approval Process

• Grant urgent access to Givinostat for all Duchenne boys over 6 — starting with those defined as broadly as possible as "ambulant", including those who can stand with support or transfer.
• Recognise that “time is muscle” — Irish boys deserve the same chance as others in Europe, the UK, and the US.
• Delays don’t just cost time — they risk boys becoming ineligible while they wait.

Duchenne doesn’t pause. Neither should the process.

 
2. Treat Duchenne Fairly — As a Rare Condition

• While only around 120 boys in Ireland live with Duchenne, entire families, schools, and communities are affected too. Their lives are full of value, and Givinostat can help them — and those who love and care for them to live better, for longer.
• The health system must adapt for rare, high-urgency conditions.
• This isn’t about jumping queues — it’s about building a fairer process for rare conditions that need it most.

3. Define “Ambulant” in Real-World Terms

The EMA limited approval to “ambulant” boys — but what does that really mean?

“Ambulant should include boys who can still stand with support — enough to transfer to a toilet, maintain dignity, and hold on to independence.”
— Duchenne UK

We ask that Ireland adopt a broad, flexible definition of “ambulant” — not the rigid trial criteria used only to measure outcomes, not to reflect real-world benefit.

 
4. Recognise That Givinostat Transforms Lives

Givinostat is not a cure — but it’s a lifeline. It preserves:

• Independence
• Dignity
• Emotional wellbeing
• The ability for families to continue caring at home, together

It gives our boys more time. More strength. More control over their lives.
And it gives us - their families - more time to hold them, support them, and fight for them.

This condition takes so much from them. For once, we can give something back.

 
✍️ Please Sign & Share

This is our moment to show that Ireland is behind these boys.

📢 Let’s make sure no Irish Duchenne boy is left behind by red tape or timelines — not when we finally have a treatment that can help.

Every delay steals a little more of their strength, freedom, and future.

Time is muscle. Don’t make them wait.