The Hyper Blog

Read the latest news and insights from the world of life science!

HRS Press Releases

View Our Latest News
Zolgensma: The "Revolutionary" Gene Therapy for Children with Spinal Muscular Atrophy

Baby's hand holding an adult's finger

Spinal muscular atrophy (SMA) is a genetic condition that results in weakened muscles and difficulties with movement. There are several different types of SMA, but the most common - and most severe - is type 1, which manifests during the first six months of a child's life. The condition progresses (gets worse) over time, and babies with type 1 spinal muscular atrophy rarely live longer than two years.

But a gene therapy called Zolgensma may be able to change that. Zolgensma is manufactured by Novartis Gene Therapies, an American biotechnology company, and while the list price is almost £1.8 million, the NHS has negotiated an undisclosed discount and infants in the UK can now receive this potentially life-saving treatment for free.

Yesterday, it was reported that a baby boy from London had become the first patient in the UK to receive this treatment. "Baby Arthur is just five months old," wrote the BBC's medical editor Fergus Walsh. "He has no way of knowing the treatment he is receiving in his right arm is the world's most costly medicine."

SEE ALSO: What is Gene Therapy?

How Zolgensma fights spinal muscular atrophy (SMA)

Spinal muscular atrophy usually occurs because there is something wrong with a gene called SMN1. Zolgensma works by replacing the faulty SMN1 gene with a healthy copy of the same gene. This change enables the patient's motor neuron cells to produce the protein needed for muscles to function properly.

Zolgensma is now available at specialist NHS centres in London, Manchester, Sheffield and Bristol. Sir Simon Stevens, the Chief Executive of NHS England, had this to say:

"It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS. The NHS Long Term Plan is committed to securing cutting-edge treatments for patients at a price that is fair to taxpayers."

Looking for gene therapy jobs?

Zolgensma is just the latest example of the enormous potential of gene therapies. The use of DNA to fight disease can produce - indeed, has produced - astonishing results that would have been unthinkable until quite recently.

If you're looking for work in this life-changing field, Hyper Recruitment Solutions can help. Click the link below to browse a wide range of vacancies from some of the UK's top biotechnology employers.

Gene Therapy Jobs >

Photo from Pixabay