'Life-changing' drug for local boys with DMD

A mother of two local boys with Duchenne Muscular Dystrophy (DMD) has welcomed their access to a new "life-changing" drug which is expected to slow down progression of the rare genetic disorder.

Athlone woman Bianca Benson has described the regulation of the drug, Givinostat, as "a cause for hope" on what has been "a difficult journey" for her two sons, Noah (12) and Rowan (10) Benson-Gately, who were diagnosed with DMD in January 2020.

DMD is a genetic disorder characterised by progressive muscle degeneration and weakness, and, according to Bianca, the approval for use of Givinostat will lengthen six months of physical deterioration out to a two-year period.

The quest for regulation of the drug for use in Ireland culminated in June when it received approval for use. Both Noah and Rowan will now take Givinostat on a twice-daily basis, improving their quality of life.

Together with her husband, Sean, and other DMD parents, Bianca has been campaigning for the drug's approval since May of last year when she first became aware of its availability and the potential benefits it could have for the lives of her two sons.

In the intervening period, the Dysart-based parents have raised the issue with Government TDs, HSE officials and clinicians. Now that approval has finally been granted, Bianca said it brought feelings of relief and joy.

"We have been waiting for this day to arrive, and that sense of elation both Sean and I felt when we received news on Friday that Noah and Rowan had been approved for the drug was incredible," Bianca said.

Both Noah and Rowan had to fulfil a number of criteria to be approved for the use of Givinostat.

"They must be able to stand independently, be over the age of six and taking steroids and also not on invasive ventilation. Thankfully, the boys meet these criteria," Bianca said.

As part of the rigorous drug approval process, the boys travelled with their parents to Temple Street Hospital in Dublin on Tuesday of last week where they had their bloods taken, each had an ECG, and they were put through physical examinations including a mobility test.

"We were in Temple Street with eight families, all of whom had children going through the examination process. It was only on Friday last that we received the phone call every parent of children with DMD wants to receive.

"It was just an emotional moment, a feeling of pure joy. Sean and I were ecstatic with the news," Bianca added.

"The boys will continue to have their bloods taken every two weeks for the next eight weeks to ensure their tryglycerine and platelet readings are at a level that can tolerate taking Givinostat," Bianca said.

Bianca is grateful that the drug will be administered "to those who need it most" following its approval in June. She said the HSE will also cover the cost of its use in Ireland.

Noah and Rowan return to Feevagh National School in Ballinasloe next month and Bianca said, they will do so knowing that there is "a brighter future ahead".