A baby with a devastating genetic disorder has become one of the first NHS patients to get a potentially lifesaving therapy.

Angel

 

A baby with genetic disoder

A five month old boy from London has become one of the first children to be treated with a groundbreaking new gene therapy after it was made available on the NHS.

At a cost of nearly two million pounds per patient sold. Genzyme is the world's most expensive drug and can increase life expectancy by as much as 40 years. 

This is the world's most expensive medicine. The list price of sojourns is almost one point eight million pounds, though the NHS has negotiated a confidential discount, which will enable dozens of affected infants to be treated each year. Five month old Arthur is one of the first to benefit. 

He has spinal muscular atrophy Esmay, a genetic condition which causes progressive muscle weakness. Without treatment, those affected rarely live beyond the age of two. Arthur is unable to lift his head and struggles to move his arms and legs, but this single infusion should prevent further loss of nerve cells.

Around one in every 10000 babies is born with spinal muscular atrophy. What is remarkable is this one off treatment may permanently stop any further decline in Arthur's muscle function. So how does sojourns mill work? It contains a healthy copy of a missing or faulty gene called Smen one, this is inserted into a harmless virus in the body. 

The virus delivers the replacement gene into the nucleus of motor neuron cells. This is essential to prevent those cells from gradually dying. The now healthy motor neuron cells start producing the missing smen protein, which is vital for muscle function. Everleigh, in a children's hospital in London, is one of a handful of centers in the UK which will offer this groundbreaking gene therapy.

Some other infants treated with sojourns may have seen their condition not just stabilize but improve. Arthus parents say they know it's not a cure and they're simply delighted that he's been treated. 


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