Boy With Muscular Dystrophy Writes Heartfelt Note to Scottish Leader
Michael Young, 9, wrote a letter to Scottish first minister Nicola Sturgeon, urging her to help make the drug Translarna available for people with muscular dystrophy.
Young has Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. He relies on Translarna, and his family is campaigning for the NHS to support the use of the drug, currently available in other countries in Europe, reports BBC.
My name is Michael Young (age 9) and I have Duchenne muscular dystrophy. I NEED your help. I have medicine called Translarna. Some boys have the same problem as me but don’t get the medicine. We need (you and me) to help Duchenne boys, because it is not something that a lot of people know about and the boys need medicine. If my medicine is stopped I will need a wheelchair. Please can I meet with you so that you can help boys to keep walking and get Translarna. I can meet you anytime and anywhere.
Translarna is currently being evaluated by the Scottish Medicines Consortium (SMC), and though Sturgeon said she could not assist with the process, she did meet with Young on Jan. 28. Sturgeon said in a statement that it was an “absolute delight” to chat with Young, adding, “He’s a really impressive and brave young man campaigning not just for himself but for other young boys with Duchenne.”
Sturgeon explained the approval process “is undertaken by experts and has to be entirely independent of Government and Ministers,” adding that the pharmaceutical company’s delay and pricing was holding things back. The SMC is expected to make a decision regarding the use of the drug in March.
“We are delighted that Michael was able to deliver his amazing, heartfelt letter to Nicola Sturgeon in person, an opportunity that doesn’t come every day,” Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said on the organization’s website. “The meeting means a great deal to the Youngs, and other families who live with Duchenne muscular dystrophy across Scotland. It is wonderful that the first minister gave up her time in support of Michael, and we hope their meeting will lead to positive discussions with the Scottish Medicines Consortium about Translarna.”