'Life-changing' therapies to be routinely available on NHS

‘Life-changing’ muscle-wasting condition spinal muscular atrophy (SMA) therapies are to be routinely available to children on the NHS.

Sharon Hodgson (c) UK Parliament

Sharon Hodgson (c) UK Parliament

The nusinersen and risdiplam treatments could help children live years longer and attend primary school.

Public health Minister, Sharon Hodgson, said: ‘Spinal muscular atrophy can have a profound impact on every aspect of a person's life, particularly for children and young people.

‘Today's draft guidance is welcome news for families across the country and represents an important milestone in improving access to innovative NHS treatments for rare diseases.

‘Drawing on both clinical evidence and the real-world experiences of patients already receiving treatment, this decision will help ensure more people with SMA can access the care and support they need to live fuller, more independent lives.'

A SMA REACH UK study found the therapies have helped 73 children with the severe form of SMA (Type 1) survive to aged five or older – more than half of those treated.

Type 1 SMA is typically diagnosed in babies less than 6 months old, and left untreated, the condition is usually fatal before the age of two. 

It is estimated that approximately 70 babies are born with SMA each year in the UK, and that there are around 1,150 people living with SMA types one, two or three in England.  

 

 

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