Spinal muscular atrophy: Parents hope to raise £1.6m for treatment

The parents of a baby with a rare genetic disorder are aiming to raise money for the "most expensive drug in the world" to try and prolong his life.

Seven-month-old Marley Powell from East Yorkshire was born with type 1 spinal muscular atrophy (SMA).

Most babies with the condition, which causes muscle weakness and affects movement and breathing, do not live past two years without intervention.

Marley's family hope to raise £1.6m to take him to the USA for treatment.

The newly developed drug is called Zolgensma and uses gene therapy to minimise the effects of the illness.

Marley's current NHS treatment - called Spinraza - ends in six months' time and there are no more UK options if he has not improved by then.

His mother Rosie-Mae Walton said: "With this Christmas being a chance of Marley's only Christmas if he does not improve, we have to try to make it as special as we can, as we do every day of his life, as we don't know what could come next."

A press photographer hopes to boost the fundraising effort by climbing England's highest mountain as many times as possible in 24 hours.

Joe Giddens, 33, will spend this weekend going up and down the 3,209ft (978m) high Scafell Pike in the Lake District.

Mr Giddens, who works for PA Media, aims to scale the mountain nine times, which would be the equivalent of climbing higher than Mount Everest.

Zolgensma has been described in the British Medical Journal as "the most expensive drug course of treatment ever" .

An appraisal by the The National Institute for Health and Care Excellence is expected to be published in March 2021.

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