Our little boy Noah has recently been diagnosed with Spinal Muscular Atrophy (SMA), a rare genetically inherited neuromuscular condition. It causes progressive muscular weakness and loss of movement due to muscle wasting (atrophy). This may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing.

Although there is currently no cure for SMA, there is the first (and currently the only) available treatment for SMA called Nusinersen.

On June 1st 2017, the European Commission approved Nusinersen for marketing under its brand name SpinrazaTM as a treatment for those with 5q SMA16. This includes all SMA Types.

Unbelievably (and in my opinion and many others including senior health professionals here in the UK) disgracefully, unethically and morally wrong, NHS England and NICE have decided not to take the recommendations of the European Medicines Agency and follow other EU countries, such as France, Germany, Italy and also in the US and Canada to make the treatment available to all. But instead to limit the number of patients by imposing its own criteria, along with what can only be described as a postcode lottery.

Despite continued pressure on the current Health Secretary Jeremy Hunt, no action, no urgency and no sign of a NICE decision is expected for at least 18 months, time my little boy just doesn’t have.

We have taken the brave decision to seek treatment outside of the UK, in Europe, but unfortunately this has come at a price.

We, our family and friends are working hard to raise the funds to enable our little boy to receive this treatment until the UK does the right thing and makes this treatment available to all. We have already raised funds to cover the initial loading doses of Nusinersen and Noah has received the treatment successfully, the improvement is already visible and his strength is improving literally every day.

With continued treatment our little boy has every chance of leading a normal life, a life of opportunities, adventures and the ability to reach his full potential.

Without treatment, he faces a life of restrictions, not only will he never be able to walk but he will lose the ability to eat orally, breathe without assistance, sit without support and will face a life of corrective surgery and ever increasing muscle weakness.

No child should have to face this.......especially when there is a treatment available which is being denied on cost alone rather than its effectiveness, which is actually outperforming what the drug company predicted.

We are an honest, hard working family, who have never asked for anything before but we need your help. Any donation would be welcomed to help us achieve our target.

If you would like to find out more about Noah or SMA then please visit our website www.helpfornoah.co.uk, here you will also find links to the main UK charities and the latest information.

Richard, Naomi and most importantly Noah X