Hastings family of toddler with life-limiting condition call for specialist drug to be more widely available

Elsie-Rose Nugent
Elsie-Rose Nugent

The family of a three-year-old girl with a life-limiting condition are calling for a drug used to treat the illness to be more widely available for sufferers.

Little Elsie-rose Nugent, of Amherst Road, was diagnosed with spinal muscular atrophy (SMA) Type 1 in September 2014 after she and her family were involved in a car crash.

The condition causes severe muscle weakness, which can result in problems moving, eating, breathing and swallowing.

Elsie-rose’s mum, Natasha Kelly, said her daughter started injections of the drug, nusinersen, marketed as spinraza, last week at Evelina London Children’s Hospital.

She said: “Us as a family have always wished for a cure for Elsie-Roses’s condition. Last December spinraza was approved in the USA.

“It is a highly specialised type of medicine that can increase the production of SMN (survival motor neuron) protein, which patients with spinal muscular atrophy lack. It then produces more protein so the motor neurones do not die, which then enables more movement.

“Here in the UK currently spinraza is approved by the European Medicines Agency (EMA) but is waiting for NICE (National Institute for Health and Care Excellence) and the NHS for their approval.

“Until then some hospitals have been granted as centres to provide it under an early access program (EAP).

“But there are around 100 children waiting to receive spinraza in the UK and some families are travelling to Paris, costing them around £3,000 a visit because they cannot get the drug here, even though their hospital provides it.

“Three children of whom we know of have died since last December waiting for the drug. The drug is free while on EAP. If there were more hospitals on EAP, it would put less strain on the NHS as more children could be treated for free.”

She said she knows a six-year-old girl with Elsie-rose’s condition, who is now able to sit up and hold her head after having several injections of the drug.

To follow Elsie-roses’s journey visit www.facebook.com/smatype1.

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