24/01/2026
On the week of his 61st birthday, Nigel Miller visited the Cardiff Haemophilia Centre at the University Hospital of Wales to be treated with Hemgenix (etranacogene dezaparvovec), a new gene therapy prescribed for the effective treatment of haemophilia B.
The new treatment marks the end of years of weekly injections for Nigel, and a life scheduled around visits to hospitals for unplanned bleeds and damage to his joints.
Nigel, who lives in Carmarthenshire, was diagnosed with haemophilia B when he was just eleven months old. As a baby, he once banged his mouth on the bars of his cot, and as the wound wouldn’t stop bleeding, Nigel’s parents knew they had to take him to see a doctor.
Previously known as the ‘Christmas Disease’ (the first patient to ever be diagnosed was a boy named Stephen Christmas), haemophilia B prevents a person’s blood from clotting properly, meaning minor injuries can lead to severe and sometimes life-changing damage.
“A simple knock, a twisted ankle, or sometimes no obvious injury at all can lead to bleeding into my joints or muscles.”
As blood leaks into joints, it can cause them to deteriorate much faster than they typically would, which can result in loss of function.
“It eats away at the cartilage and bones, and, over time, it limits the range of movement”.
As a child, Nigel’s condition was treated with fresh frozen plasma, a time-consuming process which required him to be on a drip for between four and eight days at a time, usually twice a month. Then, at the age of twelve, a newer treatment via injection, known as concentrated factor IX, became available. This is an infusion which replicates the clotting factor that those with haemophilia cannot produce.
“I started doing these injections myself at home about twice a month from the age of fourteen whenever I had a bleed.”
Until now, these injections have remained the only treatment option available and have limited the number of times Nigel has had to visit a hospital, yet his condition has continued to remain at the forefront of his mind.
“It shapes every decision, every plan, and every ordinary day of my life. From the outside, it can look normal. On the inside, it is measured in risk, caution, pain, and what others might think of as resilience.”
The new treatment, Hemgenix, introduces the gene responsible for producing clotting factor IX via the liver, preventing excessive bleeds on a permanent basis and freeing Nigel from weekly treatment.
“This is not a cure in the traditional sense, but it means I don’t have to inject myself weekly anymore. Instead of repeatedly replacing the missing clotting factor, gene therapy gives my body the instructions it has always lacked — enabling it to produce factor IX itself.”
Despite his condition and the limitations this has placed on his day-to-day life, Nigel recognises that it has encouraged him to view his circumstances with greater appreciation and pursue his aspirations with even more vigour.
“I have a wonderful wife, two loving children and a beautiful granddaughter, and have had a successful career working in the NHS. I was awarded a Fellowship by the Royal College of Speech and Language Therapists and was honoured to receive an MBE for services to people with learning disabilities in 2019.”
Living with haemophilia has taught Nigel a lot, particularly in the years leading up to now, where managing the illness has remained an integral part of his daily experience.
“People with haemophilia learn resilience early. We learn patience, planning, and how to keep going even when our bodies are saying no. There is no denying there is a constant background awareness that this condition never switches off.”
This new form of treatment, however, has given him the promise of a life not entirely governed by the management of his condition.
“I am hopeful — not for perfection, but for freedom from constant injections, fewer, if any, bleeds, less joint damage, and a life that is not planned around my condition. I imagine a future where haemophilia is something I can manage in the background.”
He remains cautiously optimistic, however, and acknowledges the significance of being the first person in Wales to receive this life-changing therapy.
“This is pioneering treatment. It comes with unknowns and long-term questions. Being first is an honour, but also a responsibility. I am deeply aware that my experience will help inform what comes next for others in Wales and beyond.”
For Nigel and many others, Hemgenix represents a major shift in the limitations placed on their lives by illness, and he takes great pride in being a part of its introduction in Wales.
“Most of all, I feel proud — proud of how far treatment has come, proud of the clinicians and researchers who made this possible, and proud to stand at a moment where living with severe haemophilia B may finally begin to look very different.
“This is not just a personal milestone. It is a glimpse of a future where inherited conditions like mine no longer define the limits of a life.”
