27/08/2025
Grandmother's Story: Fighting My MF and Advocating for the Rights of Children with Duchenne Muscular Dystrophy
For more than 35 years, I have lived with a rare skin disease called mycosis fungoides (MF). It was not until two years ago that doctors diagnosed me with follicular mycosis fungoides T-cell lymphoma. Yesterday, they offered me a stem cell transplant—the only treatment with a chance for long-term remission or a cure.
However, the transplant is very risky. Approximately 20–30% of patients die from complications, such as infections or immune reactions, even in modern hospitals. Despite this, the treatment is approved in Europe and the USA, because there are no other options for advanced MF that could offer a potential cure. In this situation, doctors and regulators accept the risk, as without it, the disease would progress and could be fatal.
Observing my own health struggle has given me a unique perspective on another fight—one that concerns children. My grandson William suffers from Duchenne muscular dystrophy (DMD). This fatal genetic disease progressively destroys muscles and robs a child of the ability to walk, run, and even breathe properly.
William has hope in a new gene therapy called Elevidys, which could halt the progression of DMD and give him a chance at a longer and more fulfilling life. Unlike my transplant, however, Elevidys is not approved in Europe. The therapy is extremely expensive, and regulators claim there is not enough long-term data for its approval, even though it is approved in the USA. This puts families like ours in a heartbreaking situation—we must raise millions of euros just to access a treatment that could save our children's lives.
The contrast is clear:
A life-threatening disease in adults has access to a risky treatment because there is no alternative.
A life-threatening disease in children does not yet have access to a potentially life-saving therapy because it is too new.
I am sharing my story not for sympathy, but to raise awareness. We must support children like William who are facing a ticking clock against DMD, and advocate for the approval and availability of treatments that can give them life and hope.
