THE family of a two-year-old girl from Oxfordshire living with a rare genetic disorder are battling for a drug to be made available on the NHS, which they say will dramatically improve their daughter’s quality of life.

Esmè Needham was diagnosed with the rare muscle wasting condition SMA type 2 (spinal muscular atrophy) in June 2017.

The condition has left the toddler without the ability stand, sit-up, or eat without assistance.

Desperate parents James Needham and Stacie Swell from Banbury say a drug Spinraza could drastically improve her condition, for which there is no cure.

Oxford Mail:

Esme Needham with mum Stacie, brother Kyle and dad James in her bedroom with the special bed and some of the kit she needs. Picture: Ric Mellis

However, health bosses this month decided against supplying the drug, also called nusinersen, to families on the NHS.

Mr Needham, 37, said: “The condition affects every aspect of her life.

“She can’t brush her own teeth, she will never walk, she can’t even hold her own head up without support.”

He went on: “The drug would be life-changing for her.

“It would give her more movement, more strength.

“For a child who can’t even hold her own head up or pick up a fork; to give her that small bit of independence, it would be such a difference for her and for us.”

SMA affects the nerves in the spinal cord controlling movement.

This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.

Mr Needham said: “Colds are really quite dangerous because she can’t clear her own throat.

“We’ve got a machine to inflate and deflate her lungs

“It’s full-on.”

The National Institute for Health and Care Excellence (NICE) ruled earlier this month that, while recognising the drug has been shown to improve a range of outcomes important to patients, it could not recommend the drug for routine use on the NHS because of uncertainties over its long-term effectiveness and its extremely high cost.

However NICE has also said it is hoped that further negotiations could be held between with the drug company and NHS England in the future.

The drug, which is the first treatment to address the cause of motor neurone degeneration in SMA, is available in many countries around the world including the USA, Japan, Norway and, for patients with the most severe form of SMA, Scotland.

According to NICE, it is estimated there are there are between 1,200 and 2,500 children and adults in the UK living with SMA.

Mr Needham said: “How something so obvious, that can help kids in countries that aren’t as financially stable as we are – how are they giving it to their kids and we can’t give it to ours?

“It’s unbelievable."

He added: “We are always going to be there to help her as much as we can but the powers that be can’t help us to help her.”

The family are now joining other parents in a fight to put pressure on health chiefs to reconsider the decision on Spinraza.

An online petition has been launched on the website while the family are planning to join a protest at the NICE headquarters in Manchester next month to put pressure on decision makers.

The family are also hoping to raise £26,000 for a new powered wheelchair for Esmè with the toddler due to start nursery in September.

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