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.
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.