A number of UK clinicians have today published an open letter calling for access to Spinraza treatment.
The letter, signed by 30 leading neuromuscular experts and clinicians from across the country, urges the health authorities to make the spinal muscular atrophy (SMA) drug available on the NHS.
NICE, the UK health regulator, has previously refused to recommend the drug because of uncertainties over its long term effectiveness and high cost.
However, leading clinicians argue that Spinraza – which is already available in 24 European countries, including Scotland – is an effective treatment.
They say trials have shown promising results, with a number of children living longer, learning to speak and crawl and even stand after treatment.
SMA is a genetic condition that makes the muscles weaker and causes significant problems with movement, getting progressively worse over time. At current, there is no cure, but there are treatments to help manage the symptoms.
There are four types of SMA, ranging from type 1 which develops in babies less than six months old to type 4 which affects adults and usually only causes mild problems.
“As physicians, there is nothing more frustrating than knowing a medicine that can help your patients is agonisingly out of reach,” the letter reads.
“For parents, hope is taken away, causing unimaginable heartbreak. As the condition progresses children are unable to regain lost function. Delay in treatment leads to the worst outcome: early death.”
The letter was published ahead of ‘The Big SMA Protest’ on 06 March, which called for UK SMA families, their friends and supporters to urge the Government to make the drug available.
The letter, first published in The Guardian newspaper, can be found here.