NHS to offer ‘world’s most expensive’ drug to cure baby spinal disease

Babies born with a genetic disease that leads to a life expectancy of only two years without intervention will now be cured on the NHS with a new £1.8 million drug.

Infants with severe spinal muscular atrophy, which causes muscle weakness leading the breathing problems and paralysis, will now be able to take the gene therapy drug Zolgensma, which has been approved for NHS use by treatment watchdog The National Institute for Health and Care Excellence.

Around 65 babies each year are born in England with spinal muscular atrophy, which is the leading genetic case of death in children.

The disease causes a defect in the SMN1 gene, which makes essential protein and enables spinal nerves in the spinal cord to control the movement of muscles.  The most severe form of the condition, Type 1 SMA, can develop in infants less than six months old, with a 90 per cent chance of dying before the age of two if left untreated.

Zolgensma has been proven in studies to help sufferers sit, walk and crawn, and prevents them from being put on a ventilator.

The one-off infusion uses a harmless virus to deliver a healthy version of the gene.  It passes into the nerve cells to replace the defective gene and restores normal function, allowing the baby to live a healthy life with minimal symptoms.

However, only babies who have a family history of SMA will be tested and campaigners are calling for all newborns to undergo checks diagnosing conditions.

Laurent Servais, professor of paediatric neuromuscular disease at the University of Oxford, said “I have seen too many families devastated by this disease, but we now have treatment options.

“However, waiting until the onset of symptoms is still too late.  Every day we delay in finding and treating these infants, we could be responsible for a child spending their life in a wheelchair.”

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Posted by Tony May, Partner/head of Clinical Negligence Department, Chadwick Lawrence LLP (tonymay@chadlaw.co.uk ), medical negligence lawyers and clinical negligence solicitors in Huddersfield, Leeds, Wakefield and Halifax, West Yorkshire.

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