There is a documentary called Matter of Time, built around two sold-out solo concerts Eddie Vedder played at Benaroya Hall in Seattle in October 2023, with the proceeds going to research into a disease most people have never heard named. Between the songs the film does something rarer than a benefit concert. It explains, plainly, what epidermolysis bullosa does to a child, and it lets you sit with the families who live inside it. The phrase that recurs in the EB community is that it is "the worst disease you've never heard of." The film's wager is that it should now be called the worst disease we will find a cure for. The science behind that wager is real. The question this piece is about is whether an Irish child gets to benefit from it, and how long they will be made to wait.

What the disease actually is

Skin is held together by a kind of biological stapling. The outer layer, the epidermis, is fastened to the layer beneath it by structures called anchoring fibrils, and those fibrils are built mostly from a protein called type VII collagen. One of the doctors in the film puts it as simply as it can be put: the protein makes a staple, the staple keeps your skin intact, and if any link in that chain is broken your skin does not stay on. Epidermolysis bullosa is the name for a group of inherited conditions where that machinery is faulty from birth. Epidermolysis means breakage. Bullosa is the Latin for blister. Put together it means fragile, blistering skin. Children born with it are called butterfly children, because their skin is as delicate as a butterfly's wing.

There are four main types, defined by which protein fails and at what depth the skin comes apart (DermNet, British Journal of Dermatology consensus classification). EB simplex, the most common and usually the mildest, comes from faults in the keratin genes KRT5 and KRT14 and the skin splits within the surface cells. Junctional EB involves laminin-332 or collagen XVII and the skin separates at the junction between the layers. Kindler EB, the rarest, with only a few hundred cases ever reported, comes from the FERMT1 gene. The fourth type, dystrophic EB, is the one the film is mostly about, and it is caused by faults in a single gene, COL7A1, the gene that carries the instructions for type VII collagen (review of EB genetics, PMC).

The recessive form

Dystrophic EB comes in two inheritance patterns, and the difference between them is the difference between a hard life and a brutal one. The dominant form is generally milder. The recessive form, recessive dystrophic epidermolysis bullosa, or RDEB, is the severe end of the entire EB spectrum.

Recessive means a child has to inherit two faulty copies of COL7A1, one from each parent, to be affected (DermNet). The parents themselves usually carry one faulty copy each and have no symptoms at all. They are healthy, the genetics are silent, and then a baby arrives with skin that blisters and tears from the first touch. In the film one mother describes the day her daughter was born as the best day of her life, and the day a doctor quietly said the words "epidermolysis bullosa", and "it would be for life", as the worst. With two broken copies of the gene the body makes little or no working type VII collagen, the anchoring fibrils never form properly, and there is nothing stapling the skin together.

What follows is lifelong and it gets worse with time, not better. Blistering and open wounds from minor friction. Healing that lays down scar tissue, which over years fuses fingers and toes together into what clinicians call mitten deformity, or pseudosyndactyly. The same fragile lining runs through the mouth and the oesophagus, so swallowing scars and narrows, which brings on malnutrition and growth failure. Many children are fed through a tube into the stomach. There is chronic anaemia, eye and dental involvement, constant pain, and a daily ritual the film does not look away from: hours-long bandage changes, and bleach baths to soak the dressings off and hold infection back. As one parent says, nothing in that ritual treats the disease. It only wraps the patient and tries to protect them from the next injury.

Then there is the part that makes RDEB not just a disease of suffering but a disease of shortened life. Skin that is endlessly wounded and endlessly healing eventually turns. People with severe RDEB develop an unusually aggressive form of skin cancer, cutaneous squamous cell carcinoma, arising in their chronic wounds, and it is the leading cause of death in early adulthood. The cumulative risk is staggering: in the severe generalised form, by their mid-thirties most patients have had at least one such cancer, and by their mid-fifties the figure approaches ninety per cent (DermNet; 28-year retrospective study, Acta Dermato-Venereologica). These tumours are multiple, they spread early, and survival after diagnosis is often short. A child with RDEB is, in a real and measurable sense, running a clock.

For the first time, medicine that addresses the cause

For most of the history of this disease the clock ran unopposed. A decade ago the standard of care for EB was, in a researcher's words in the film, primarily just asymptomatic care. Bandages and wound dressing. Nothing reached the genetic fault underneath.

That has changed, and it has changed fast, which is the reason the film exists. The first shift is symptomatic but real. Filsuvez, made by Chiesi, is a topical gel derived from birch bark that helps EB wounds close faster and eases the pain of dressing changes. It is not a gene therapy. It treats the wound, not the cause. It earned European approval in 2023 and was approved by the US Food and Drug Administration in December 2023 on the strength of a phase 3 trial of 223 patients across 28 countries (Healio).

The second shift goes to the root. Vyjuvek, made by Krystal Biotech, is the first redosable gene therapy ever approved. It is a gel, applied to wounds once a week, that uses a disabled herpes simplex virus to carry working copies of the COL7A1 gene directly into the skin, so the body starts producing the type VII collagen it could not make before. It is corrective, not merely palliative, and it can be applied at home rather than in a hospital. The FDA approved it in May 2023 (FDA), and the European Commission approved it in April 2025, calling it the first corrective medicine for dystrophic EB approved in Europe (European Medicines Agency).

The third shift is aimed squarely at the recessive form. Zevaskyn, made by Abeona Therapeutics, is the first cell-based gene therapy for RDEB. Surgeons take a patient's own skin cells, the genetic fault is corrected in a laboratory, and the repaired cells are grown into sheets and grafted back onto the patient's wounds in a single application. The FDA approved it in April 2025 (Abeona; Pharmacy Times). It is the clinical-trial skin graft the film follows in the laboratory. It is approved in the United States and not, as yet, in Europe.

Three approvals in two years, for a disease that had nothing a decade ago. The doctors in the film are not exaggerating when they say the cure is no longer in their imagination but in the laboratory, the hospital and the patient's home.

The question Ireland has to answer

Here is where the story stops being only about medicine. A treatment existing is not the same as a patient reaching it. Around 300 people in Ireland live with epidermolysis bullosa (DEBRA Ireland). The question for them is not whether the science works. It is whether the Irish system will let them have it, and how long the waiting takes.

The answer so far is sobering. Filsuvez, the wound gel, was approved across Europe in 2023. The HSE agreed to reimburse it in Ireland only in May 2026 (RTÉ News). That is roughly three years for a symptomatic treatment, and it was reported as a hard-won victory. DEBRA Ireland's chief executive called it "a long journey" and "a transformative moment". Three years, for a gel.

Vyjuvek, the corrective gene therapy, the one that actually addresses the genetic fault, was approved in Europe in April 2025. It is not reimbursed in Ireland. DEBRA Ireland's own estimate is that "it could still be between seven and 12 years before we see Vyjuvek available to people here" (DEBRA Ireland). Zevaskyn, the graft built for the recessive form, is not yet approved in Europe at all.

This is not a freak delay. It is the system working as it ordinarily works. Ireland sits in the lowest quartile in Europe for introducing new medicines, and the reported average gap between European approval and Irish reimbursement is around 1,024 days, against roughly 611 days elsewhere in Europe (RTÉ News). Close to three years, on average, between a medicine being judged safe and effective for Europe and an Irish patient being allowed to have it. For most conditions that delay is an injustice measured in suffering. For recessive dystrophic EB it is measured against the cancer clock, where the difference between treatment now and treatment in seven to twelve years is, for some patients, the difference between a treatment and a post-mortem.

The other half of the barrier is price, and it is worth saying the number out loud. Vyjuvek launched in the United States at a list price of 24,250 dollars per vial, which works out at an estimated steady-state cost of around 631,000 dollars per patient per year, with one analysis putting the lifetime cost as high as 15 to 22 million dollars per patient (Drugs.com; JAMA-published spending estimate, PMC). Those numbers are why the reimbursement question is hard, and they are also a choice. A price is set by a company and negotiated by a state. The science that produced the molecule was funded substantially by a patients' charity, which brings the story back to where it started.

Who funds a cure, and who delivers it

The film's benefit concerts feed the EB Research Partnership, founded in 2010 by a group of parents along with Jill and Eddie Vedder, who got involved when they learned that the child of one of Jill's childhood friends had the disease (EB Research Partnership). The organisation runs on a model it calls venture philanthropy: when it funds a research project it retains an upside, so that if a therapy reaches the market the returns flow back into funding the next piece of research. By its own account it has raised more than 80 million dollars and helped fund the first approved gene therapy for EB. It is a serious, and genuinely clever, answer to the problem of paying for research into a disease too rare for an ordinary market to bother with.

That model got the science done. It cannot do the second half. A charity can fund the discovery of a cure. It cannot make a national health service reimburse it, and it cannot on its own bring the price within reach. That part is a public decision, made by states, agencies and the companies that set the prices, and it is the part where Ireland is slow. The cruelty of EB is written into a gene. The wait an Irish butterfly child now faces for the medicine that answers that gene is written into policy, in reimbursement timelines and pricing negotiations and the ordinary, unhurried machinery of who gets approved when.

The film is called Matter of Time because the people in it believe a cure is coming, and the science says they are right to. The phrase has a second meaning the film does not dwell on. For a child with the recessive form, time is exactly the thing the disease is spending. The science has done its part. Whether the system does its part, fast enough to matter, is the question Ireland has not yet answered.

Vedder is the hook here and the lowliest of stars in the most well studded event I have yet seen. This is perspective!

I promise you this; if you are a parent or even just a human being, this documentary will reach the very core of you!! Cannot recommend enough! Do yourself a favour!

Matter of Time (2025), directed by Matt Finlin, is streaming on Netflix. DEBRA Ireland, the national charity for people living with EB, provides support, advocacy and information across the country at debra.ie.

Sources