On Rare Disease Day 2025, a message from our founders, Emily and Alex
Friends, today we are sharing with you our update on the Time is Muscle campaign on what is a special day – rare disease day – the only day in the year where we celebrate the rare disease community as a whole.
Living with a rare disease often makes us feel that it’s us against the world.
And nowhere has this been brought into sharper relief, than in our Time is Muscle campaign.
Alex and I desperately wish that we could mark this day by sharing good news of patients getting givinostat through the Early Access Programme (EAP).
But, maddeningly, this is still not the case.
We thought it would be easy. In November we discovered that ITF Pharma UK was offering its drug givinostat FOR FREE to eligible patients. And so we told patients the eligibility criteria, and to talk to their consultant, and to write to their NHS Trust to get it up and running. Since then our community has been remarkable, helping secure a broad label for givinostat from the MHRA, and culminating in a parliamentary event during which, remarkably, the Health Secretary Wes Streeting, promised to help our Time is Muscle campaign.
We thought it would be relatively easy to fix: company offers drug for free, NHS delivers it. But the challenges are huge.
We’ve learnt that;
- Clinics have found it hard to secure the additional resources needed to do blood tests
- Hospitals have struggled to find the space – and the additional staff time – in pharmacies to store and dispense the drug
- Taking part in the EAP requires extra nurse support, which the clinics don’t have enough of
- Capacity in phlebotomy (blood) services has been an issue for some clinics
- Ethics approval (before the MHRA licence in December) held up many applications to take part in the EAP before Christmas
- The wait for internal Trust governance procedures (such as approval from drug and therapeutic committees) has been slow
But we are not giving up. All of these problems can be solved. And we won’t stop until everyone who can benefit from givinostat can access it.
- We are continuing to push parliamentarians to persuade Trusts to take part in the EAP
- We are engaging with the Government and the Department for Health and Social Care to see if they can provide extra resources for clinics
- We are working with the NorthStar clinical network to educate the doctors on why this is important to our community, and to learn more about the barriers and challenges they are facing
- We will keep writing to the CEOs of all the NorthStar hospitals – our latest letter was last week, and we hope to meet with some of these CEOs during March
- We have submitted two FOIs to all the NorthStar hospitals about the EAP, and about other schemes like the EAP
With love,
Emily Reuben OBE, Co-founder and Chief Executive of Duchenne UK
Alex Johnson OBE, Co-founder of Duchenne UK and Chief Executive of Joining Jack