Spinal muscular atrophy: Spinraza approval delights family

The mother of a girl with a muscle-wasting disease is delighted a drug that may slow her daughter's condition has been approved for use on the NHS.

NHS England said it has agreed the use of Spinraza to treat spinal muscular atrophy (SMA).

Heidi Prescott-Booth, 11, from Wolverhampton, was diagnosed with the condition when she was three.

Her mother, Katie Prescott, said they are "over the moon" that she will be able to access the drug.

SMA is a genetic condition which affects the nerves in the spinal cord, weakening muscles and causing problems with movement, breathing and swallowing.

It can significantly reduce life expectancy when it develops in babies and toddlers.

For Heidi, the condition means she struggles to walk.

Mrs Prescott said: "We are speechless, it is incredible. When Heidi found out she just cried her eyes out."

Between 600 and 1,200 children and adults are living with the condition in England and Wales.

While not a cure, trials have shown that Spinraza, the trade name of the drug nusinersen, can slow the effects of SMA in some cases.

Already approved for use in Scotland, the National Institute for Health and Care Excellence (NICE) has previously not recommended it because of uncertainties over its long-term effectiveness and its high cost.

But NHS England said it had come to an agreement with its makers, Biogen, to fund treatment for a limited time, allowing more data to be collected on its effectiveness.

Simon Stevens, its chief executive, said: "This promising treatment has the potential to be life changing for children and their families."

The charity TreatSMA said it "cannot express" how happy it was with the decision.

The treatment will be made available to the youngest and most severely affected patients immediately.

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