Zolgensma: Marley gets 'miracle drug' for rare disorder

A boy with a rare genetic condition has finally received NHS treatment using a "miracle drug", his parents have said.

Rosie-Mae Walton and Wes Powell, from East Yorkshire, had planned to take 16-month-old Marley, who has type 1 spinal muscular atrophy, to a US hospital.

But Zolgensma, which has been described as the world's "most expensive" drug, with a list price of £1.79m, was approved for use by the NHS in March.

The pair said Marley seemed to be doing well after treatment in Sheffield.

The one-off Zolgensma treatment, which Marley received on Tuesday at Sheffield Children's Hospital, involves what could become the most expensive drug approved by the National Institute for Health and Care Excellence.

The British Medical Journal has said it is "the most expensive drug course of treatment ever" .

Ms Walton said it had been a "rollercoaster" experience since it was licensed while they waited to find out if their son was eligible.

She and Mr Powell, from Driffield, had been fundraising to help treat their son's condition in the US.

"As soon as we found out that it came to the UK, we just cried our eyes out," Ms Walton said.

"Then, when we got the antibody test and that came back negative, that he was eligible for Zolgensma, I read the email and we just cried again - we just cried all day."

Their son's condition causes muscle weakness and affects movement and breathing, meaning most babies who have it do not live past two years without intervention.

Children with the condition often need breathing assistance or a feeding tube.

The money raised for Marley will be used for continuing treatment including private physiotherapy and equipment, his family said.

Ms Walton added: "As much as he's got his treatment, it's not just going to be an easy ride. So, we just thank every single person that's helped out for that."

Tony Hart, consultant paediatric neurologist at Sheffield Children's Hospital, described Zolgensma as a "miracle drug" and said more than 30 children had now received it in England.

"One of the great things about the NHS is that decisions about costs are taken out of our hands and our priority is giving as many children who need the drug when we can, and as fast as we can," Mr Hart said.

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