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5-month-old gets wonder drug Zolgensma that costs 1. 79 million pounds per dose
British Baby Arthur Morgan is truly destiny’s child. Shortly after he was born six weeks prematurely, hopes of his survival beyond two years looked bleak. The five-month-old bonny baby was diagnosed with spinal muscular atrophy (SMA) at the Evalina Children’s Hospital, London.
And then suddenly, in the most dramatic turnaround, Arthur became Britain’s first and youngest patient to be administered a new wonder drug Zolgensma on May 25, the world’s most expensive drug, under the National Health Service (NHS) programme. It costs a whopping 1. 79 million pounds for one dose.
Once administered, Zolgensma teaches the body to manufacture a vital protein that arrests and reverses the process of SMA.
Medical expert says that Zolgensma, manufactured by American drug company Novartis has the capacity to not only arrest the degenerative effects of SMA but completely reverse the process, allowing children to lead a near-normal life. Once SMA sets in, children gradually lose the ability to sit, crawl, walk and become totally helpless on account of the massive muscular atrophy that takes place within the body.
Once administered, Zolgensma works by replacing a faulty gene that stops the body from making a protein called SMN. This protein is critical for muscle function. The corrected gene is put into a harmless virus which is then injected into the baby’s hand. It works just like a vaccine.
Once SMA sets in, children gradually lose the ability to sit, crawl, walk and become totally helpless on account of the massive muscular atrophy that takes place within the body.
The gene starts making the SMN protein which prevents the loss of motor neurons — nerve cells that help co-ordinate movement in the spinal cord.
Up until two years ago, there was no cure for this condition, which affects 60 children every year in Britain. And such children invariably never live beyond the age of two years.
Now there is every reason for hope and exultation. Arthur’s parents Reece and Rosie are ecstatic beyond words. Young Reece, Arthur’s father, was overcome with emotion and broke down several times in the hospital. He works as a plasterer.
Once administered as an intravenous drip, babies quickly gain the ability to sit, crawl and walk. Additionally, it also precludes the use of a ventilator.
Four specialist centres have been commissioned to administer the treatment, including the Evalina Children’s Hospital, London.
The other centres are the Manchester United NHS Foundation Trust, Sheffield; The Children’s NHS Foundation Trust and University Hospital, Bristol; and the Weston Foundation Trust.
Dr Elizabeth Wraig, consultant paediatric neurologist at Evalina London says: “The treatment will bring relief and hope to families affected by SMA”.
Medical expert says that Zolgensma, manufactured by American drug company Novartis has the capacity to not only arrest the degenerative effects of SMA but completely reverse the process, allowing children to lead a near-normal life.
The NHS Chief Executive, Sir Simon Steven, adds: ‘It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur through the NHS”.
Sally-Anne Tsangarides, General Manager at Novartis Gene Therapies in the UK said: ‘Novartis is delighted to be part of this remarkable milestone — it is a hugely important step for babies in England diagnosed with this devastating genetic condition and we thank all those who have been involved in the landmark agreement that has made it possible.’