A genetic condition which can kill children before the age of 2, and which has recently had funding cut for its only known drug was the subject of a recent Parliamentary reception attended by Chesterfield MP Toby Perkins.

Spinal Muscular Atrophy is a very serious genetic medical condition, which usually has an onset in childhood or at birth, progressively results in the deterioration of a person’s muscles and movement in general. It causes severe disabilities, and drastically reduces life expectancy. A child with Type 1 SMA would be unlikely to live past the age of two.

Recently, to the anger of SMA patients, the NHS was advised not to offer the only drug to treat this condition because of cost, despite the drug being potentially life-saving.

This Tuesday, the charity Spinal Muscular Atrophy Support UK held a drop in session in Westminster for MPs wishing to learn more about the illness, and what can be done to help those suffering.

There are 4 different types of SMA, each differing in severity, onset and long-term prognosis, amongst other things. Nonetheless, all the types of SMA affect each patient and their families considerably.

It is estimated that between 660 and 1320 people in the UK are living with SMA, meaning that the cohort of people who would receive the drug is a relatively small one.

Whilst the drug is not a cure for the condition, it is known to extend the life expectancy of sufferers and it is too early to say whether it might offer SMA patients the chance to live into adulthood.

There is evidence that the drug helps improve a patient’s prognosis, which is why NICE and NHS England should take urgent action to review their judgement that the drug is not cost effective.

Toby was recently approached by the parents of a child in his constituency to help after their child was diagnosed with Type 1 SMA.

Luke Catt, contacted Toby recently to ask for his support to get NHS England to offer the drug Nuisinersen to its patients. The drug is currently offered by NHS Scotland, but is much more difficult to obtain in England. NICE provided first guidance that NHS England should not offer this drug, as they say the cost is too high to be considered cost-effective.

Fortunately, Mr Catt’s daughter Grace, is one of the last children to receive the drug before the NICE guidance comes into effect in November, which offers her hope of a longer life.

But any children in England who are diagnosed after November will be denied the drug.

Doug Henderson, an SMA Support UK worker, discussed in detail with Toby what he could do as an MP to support those affected.


“There is nothing more devastating than the news that your child has a life threatening condition. To then discover that the NHS is refusing you a drug that would be available if you lived in most of the countries in Europe is doubly heart-breaking. I will be putting pressure on the Health Secretary and NICE to re-consider their decision, so that other children like Gemma might have the chance to live a longer life with SMA.”

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