March 17th 2023.
(Image Source: https://metro.co.uk) Spinal muscular atrophy leads to a gradual wasting away of people's muscles. A potentially life-saving therapy, known as onasemnogene abeparvovec and marketed as Zolgensma, has been approved by medicine regulators to address the underlying genetic cause of the disease and possibly end it permanently. The cost of the one-time treatment in the US is $2.1 million, making it one of the most expensive drugs in the world. In the UK, a baby is born every five days with this genetic disorder, which is the leading genetic cause of death in children. Five-month old Arthur Morgan was the first patient in England to receive the SMA treatment on the NHS, which was made possible by the National Institute for Health and Care Excellence. Before 2019, there were no treatment options available and parents had to raise enough money to get their child abroad for the therapy, and then pay over a million dollars for it. Another drug, Spinraza, is even more expensive, costing $4 million for a decade of yearly treatment. Theo Greenball, a one-year-old who was diagnosed with the ‘terminal’ disorder at six months, was one of the first babies to become eligible for the life-saving SMA treatment on the NHS.
Spinal muscular atrophy brings about the continuous wasting away of people's muscles. A promising 'cure' for this cruel sickness, which in its most serious structure results in the death of newborn children before they turn two, is going to be available on the NHS soon. SMA is a hereditary issue that takes away people's capacity to sit, creep, walk, talk, swallow, and breathe. Onasemnogene abeparvovec, a gene therapy more popularly known as Zolgensma, which rectifies the basic hereditary cause of SMA, has been given the green light by drug controllers, and may permanently end the inherited illness. The one-time treatment in the US is one of the priciest medications in the world, costing around £1,800,000. Every five days, a baby is brought into the world in the UK with this disease, which is the primary genetic cause of death among children.
(Image Source: https://metro.co.uk) Five-month old Arthur Morgan was the first individual in England to be given the possibly life-saving medicine on the NHS, after the National Institute for Health and Care Excellence gave its approval for the gene therapy to be used in early presymptomatic stages. Before 2019, no treatment alternatives existed. Zolgensma was only made accessible to families on the NHS on a controlled experimental basis, forcing some parents who were scared for their kid's life to raise enough money to not only fly out of the country for the treatment, but to pay more than a million for it. One of the few other medications that treat SMA, Spinraza, is even more costly, with a decade of yearly treatment easily setting you back $4,000,000.
(Image Source: https://metro.co.uk) Theo Greenhall was one of the initial infants to be qualified for the life-saving SMA therapy on the NHS.
(Image Source: https://metro.co.uk)
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