NHS announces funding for Spinraza to help children with rare muscle-wasting condition
Children living with a rare muscle-wasting condition will now benefit from a new drug.
Children living with a rare muscle-wasting condition will now benefit from a new drug.
Nusinersen, also called Spinraza, has been approved by the health watchdog NICE, meaning patients with Muscular Atrophy will be able to receive the treatment on the NHS.
What is spinal muscular atrophy?
The condition affects the nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing.
Where it develops in babies and toddlers, it can significantly reduce life expectancy.
Between 600 and 1,200 children and adults are currently living with the condition in England and Wales.
How do you contract spinal muscular atrophy?
There are several types of SMA, which start at different ages, some more serious problems than others.
In most cases, a child can only be born with SMA if both of their parents have a faulty gene that causes the condition. People with the most severe forms of SMA usually die before the age of 2.
This promising treatment has the potential to be life changing for children and their families.
The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long term benefits.
This latest deal coming on the heels of a number of other recent successful negotiations demonstrates that there is no reason for other companies not to show equivalent flexibility in order to benefit NHS patients, taxpayers and indeed themselves.
– SIMON STEVENS, NHS ENGLAND CHIEF EXECUTIVE
We are very pleased that we can now recommend Spinraza for people with SMA. The committee has recognised that Spinraza is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognised that there are significant uncertainties, particularly around its long-term benefits.
All along we have felt it important to give all parties every opportunity to try to find a way to mitigate these uncertainties in order to make Spinraza available to patients in England.
Today’s announcement shows that, where companies show appropriate flexibility, it is possible to find a way to provide important treatments to patients in a way that is cost effective for the NHS and taxpayers.
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