For now, Ann Marie Harte’s five-year-old son, Lewis, can still walk. He can no longer run, and he has a hard time with the stairs. The trampoline exhausts him, and he may never be able to play sports. This is a challenging transition for any young boy, but Harte has hope Lewis will receive the medicine he needs in time. The Irish mother worries that if the wait is too long, Lewis will require a wheelchair permanent, and may be at high risk for cardiac arrest in his early thirties.
Lewis suffers from Duchenne Muscular Dystrophy (DMD), a rare form of muscular dystrophy that predominantly affects young boys. Patients are born with a condition that disrupts dystrophin production, which leads to muscle weakness. As the disease progresses, it can cause difficulty moving, quadriplegia, and eventually cardiac or respiratory weakness. To read more about DMD, click here.
Like any mother, Ann Marie grieves over the difficult journey her son will have to take. She told the Independent that Lewis is a sweet child, and highly intelligent. She’s frustrated and devastated right now, but not only because of the diagnosis. Last month, she heard the big news she had been waiting for: Ireland’s Health Services Executive (HSE) will not fund the DMD treatment Translarna.
Translarna is the first drug approved to treat the underlying cause of DMD. Although it is new, its effects have been life-changing for afflicted patients. The key is starting treatment early. If Lewis starts the drug now, he will not return to the state he was before DMD, but the treatment can keep DMD from progressing. If he doesn’t start the treatment now, he could lose the ability to walk.
He would need a wheelchair, which could bring future scoliosis, ensuing surgeries, and a slew of other medical complications that Ann Marie feels are avoidable. Lewis has already lost the past year waiting for treatment, and his mother worries how he will fare if they wait much longer.
Families affected by DMD met with senators at a Leinster House Meeting earlier in the month. Michelle Young, a mother from Scotland, flew in to speak about her son, whose life has been changed by Translarna. His case of DMD has shown no progression since he began treatment.
HSE has released statements that after careful consideration, they will not accept the families’ applications to fund treatment. The manufacturer has appealed the issue to the High Court in Ireland, who will weigh in on the decision.
For now, Ann Marie would do anything to be able to save her son. It tortures her that effective treatment is so close, and still so far from being available. Her husband and her both work in Ireland, and their daughter is in the middle of her final year in school. It isn’t truly feasible for the family to suddenly move out of the country, but it may be the only option they’re left with.
