Mother of boys with DMD calls for 'life-changing' drug's approval

A mother of two local boys diagnosed with Duchenne Muscular Dystrophy (DMD) is calling for the regulation of a new drug which would help to slow down progression of the rare genetic disorder.

Noah (11) and Rowan (9) Benson-Gately were diagnosed with DMD in January 2020 and in the intervening time, the genetic disorder has had a profound effect on their lives and that of their parents, Athlone native Bianca Benson and Dysart's Sean Gately.

DMD is a genetic disorder characterised by progressive muscle degeneration and weakness.

There is currently no cure for the condition, which Roscommon 'Join Our Boys' twins George and Isaac Naughton are also battling.

Speaking to the Westmeath Independent this week, Bianca Benson called for regulatory approval of a drug called Givinostat to allow her two sons access it in order to curtail progression of the disorder. “We were first made aware of Givinostat in early May,” Bianca explained.

“Having researched the drug, it can only have a positive impact on the life of our two boys,” Bianca said.

According to Bianca the use of Givinostat will lengthen six months of physical deterioration out to a two-year period.

The Dysart resident called for swift regulatory action to have the drug approved, saying, "we don't have time to wait".

With representatives from Muscular Dystrophy Ireland, Bianca will travel with other Duchenne parents to Leinster House on Wednesday next, June 11, to meet with the Minister for Health, Jennifer Carroll McNeill, and impress upon her the urgent need to make the drug available to her two sons.

"Not alone will we be fighting the cause of our two sons but we will be speaking on behalf of the 110 boys in Ireland who have been diagnosed with DMD," Bianca said.

The drug is available for use in the UK, and Bianca said she was aware of a 21-year-old DMD patient who was still walking and driving as a result of its positive impact.

Givinostat has received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) for conditional approval, with a formal decision expected from the European Commission by July.

If formally approved for use, Bianca stated that it will be costly to administer for both boys.

"A one milligram dose of Givinostat costs €33.80 and our two boys would require 31 milligrams of the drug twice daily, amounting to €4,191.20. The drug will be available for boys who are six years and older and who are still able to walk," Bianca said.

While maintaining a positive outlook despite everything, Bianca said it "breaks her heart" to see the impact which DMD continues to have on Noah and Rowan.

"Noah has to use a wheelchair to when going to school and is at an age where he has now become understandably frustrated with his situation. He voices a lot of his frustrations to his Special Needs Assistant (SNA) in Feevagh NS and often asks why is he getting worse?

"When you see your son sitting in a wheelchair in the middle of a school yard it's just heartbreaking," Bianca said.

Rowan has to use a wheelchair when the family are travelling long distances, but Bianca is proud of the manner in which the boys take care of each other.

"The boys really have a very close bond and are very considerate of each other. Rowan is always there for Noah when he is in need of a hug," Bianca said.

The challenges presented by DMD are numerous, muscle weakness starting in the legs before progressing through the body and eventually negatively impacting the organs.

"The body is not producing significant amounts of the protein dystrophin that helps keep muscle cells intact restricting movement and causing chronic fatigue. Imagine trying to walk around with 10lb weights strapped to your limbs and back. That is the daily situation facing Noah and Rowan and if both take a fall in the house they need assistance to get back on their feet," Bianca added.

The Dysart community continues to rally around the family, especially those within the Noah and Rowan Benson Gately Support Group established in 2020.

"I cannot speak highly enough of our local community. They have helped to create a DMD conscious environment for Noah and Rowan and for that Sean and I will be forever grateful," Bianca said.