UK Reports 1st Treatment for Spinal Muscular Atrophy

Varieties

June 3rd, 5:56am

London - Asharq Al-Awsat

Arthur Morgan underwent gene therapy infusion at the Evelina London Children’s Hospital. (PA)

A five-month-old baby has become the first patient in England treated with a potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy, according to The Guardian. Arthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London children's hospital on May 25. Until two years ago, there were no treatment options available for children with spinal muscular atrophy (SMA), which is the leading genetic cause of death for children. But babies could have the ability to sit, crawl and walk after being treated with US gene therapy Zolgensma, which has been called the most expensive drug in the world. Zolgensma, which has a list price of £1.79m per dose, was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.' Arthur, who was born six weeks premature in December, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier. His father, Reece Morgan, 31, who works as a self-employed plasterer, said: "When we found out that Arthur would get the treatment, and be the first patient, I just broke down. It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment, as we learned about his condition and what it might mean for him and our family. We still don't know what the future will hold, but this gives Arthur the best possible chance to give him the best possible future." Babies born with Type 1 SMA, which is the most common form of the condition, experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years. Studies found that a single treatment with Zolgensma has helped babies with SMA to sit, crawl and walk, and also prevented them from having to be put on a ventilator. Dr. Elizabeth Wraige, consultant pediatric neurologist at Evelina London, said: "This treatment will bring hope to families affected by SMA who have fought so courageously against it." NHS chief executive Sir Simon Stevens added: "It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS."

from Asharq AL-awsat https://english.aawsat.com/home/article/3006736/uk-reports-1st-treatment-spinal-muscular-atrophy