A baby in England has been the first patient on the countrys NHS to receive a potentially life-saving new gene therapy to treat spinal muscular atrophy (SMA).<\/p>\n
Five-month-old Arthur Morgan was given a dose of the treatment known as Zolgensma at Evelina London Children's Hospital on May 25.<\/p>\n
Arthur was born in December last year and diagnosed with SMA, a rare and debilitating neuromuscular disease that prevents muscle development, just a few weeks ago.<\/p>\n
Babies born with SMA experience problems with movement, breathing, and swallowing.<\/p>\n
The novel gene therapy treatment, produced by US pharma giant Novartis Gene Therapies, repairs affected genes inside the cells, making it easier to manage and potentially cure the disease.<\/p>\n
But the wonder drug comes at a price.<\/p>\n
Zolgensma is one of the world's most expensive drugs with a price tag per dose of 2.08 million. Fortunately for Arthur and his family, the NHS negotiated a non-disclosed discounted price for the drug, making it more accessible for patients suffering from SMA to receive the treatment.<\/p>\n
Arthurs father, 31-year old Reece Morgan was overjoyed at the news.<\/p>\n
\"When we found out that Arthur would get the treatment, and be the first patient, I just broke down,\" he said.<\/p>\n
\"It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment, as we learnt about his condition and what it might mean for him and our family\".<\/p>\n
In a statement, Sir Simon Stevens, the chief executive of NHS England, also acknowledged the importance of using innovative therapies to treat diseases and disorders, such as in this case.<\/p>\n
\"It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS,\" he said.<\/p>\n
Getting to this moment was a long road, Imran Kausar, Medical Director at Novartis Gene Therapies UK, told Euronews. <\/p>\n
\"These are hugely transformative therapies. To bring something in that only requires treatment once, that actually requires a lot of systems and processes to change,\" he said. <\/p>\n
\"The NHS has tried its hardest to deal with the changes\".<\/p>\n
The agreement between Novartis and the NHS is part of a \"series of smart deals\" aimed to secure more cutting-edge treatments for the British people, according to the NHS website.<\/p>\n
Novartis says that while the cost of the single-dose treatment sounds expensive, it isnt when you compare the alternative treatment for SMA which can include ongoing hospital admissions, ventilators and round-the-clock care.<\/p>\n
\"Our mindset is to think of things over a long period of time,\" Kausar added. <\/p>\n
\"When you consider lifetime costs - actually gene therapies arent expensive. The value we provide is more beneficial to healthcare systems\".<\/p>\n
Whilst Kausar was reluctant to say if Zolgensma was a complete cure given the many variables, including when the infant is diagnosed and when the drug is administered, he does believe there is a curative future for gene therapy drugs \"if the entire system is in sync\". <\/p>\n
I do see us moving into a genomic era which is an era of medicine where we can harness our understanding of genetics and the impact on health in a much great way,\" he told Euronews.<\/p>\n
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More here:<\/p>\n
A revolutionary gene therapy is bringing hope to UK parents - but it comes with a steep price tag - Euronews<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" A baby in England has been the first patient on the countrys NHS to receive a potentially life-saving new gene therapy to treat spinal muscular atrophy (SMA). Five-month-old Arthur Morgan was given a dose of the treatment known as Zolgensma at Evelina London Children's Hospital on May 25. Arthur was born in December last year and diagnosed with SMA, a rare and debilitating neuromuscular disease that prevents muscle development, just a few weeks ago Continue reading