5-month-old baby Arthur Morgan has become the first in the UK to be rescued from a “miracle” medicine, which alone costs 2.1 million euros.
Five-month-old Arthur Morgan was diagnosed with spinal muscular atrophy (SMA), which usually leads to paralysis and death within two years. Last week, he became the first patient to receive Zolgensma, a new gene therapy injected into the arm after the NHS reached a historic agreement with its manufacturer.
According to The Sun., his father, Reece Morgan, 31, said: “When we found out that Arthur would be the first to get the treatment, I was shocked. It was such a distraction for a few weeks, filled with a lot of anxiety as we learned about his condition and what it might mean. This is the best treatment possible and will give him the best life he can have – and we are so grateful that the NHS is here for him. ”
Zolgensma, manufactured by the Swiss firm Novartis, is the most expensive medicine in the world. One dose is enough to stop SMAs, potentially giving babies the ability to sit, crawl and walk. It is given as a single intravenous infusion and contains a copy of the missing SMN1 gene.
Novartis bosses have defended the award, saying a single dose is more valuable than expensive long-term treatments. Experts have said that Zolgensma is a “miracle” medicine and “could be close to being a cure” if administered early enough.
Reece and Arthur Rosie's mother, from South London, had taken him to A&E after noticing that he was immobile, blurred and could no longer hold his head. About 65 babies are born with SMA in England each year. Causes muscle weakness, loss of movement and difficulty breathing.
