We are a UK initiative of parents of children affected by spinal muscular atrophy (SMA) as well as adults living with SMA who joined together to advocate for broad access to treatments for this severe neuromuscular disorder.
The first drug to treat SMA, Spinraza (previously referred to as nusinersen), was approved in the United States on the 23 December 2016 and in European Union on 1 June 2017. The authorities on both sides of the Atlantic considered the drug effective in all types of SMA. Anyone who suffers from SMA can be treated with the drug.
This however has not been the case here in the UK.
Even though Spinraza has shown remarkable results in babies, children and teenagers with all types of the disease, the limited amount of data available for the milder forms of SMA mean that we are facing a real risk that the NHS will limit offering Spinraza to the youngest children with the most severe form of the disease.
Everyone with SMA has a right to treatment.
We are campaigning to ensure that people with all types of the SMA get the opportunity to be treated. We do not believe that sitting back and waiting or hoping for the NHS to offer the drug will get it to our children or ourselves.
We intend to work with other members of the UK community of SMA patients and their families in order to advocate for broad access to Spinraza and any further therapies.
Our campaign highlights the devastating effects that spinal muscular atrophy has on sufferers and shows how the treatment has improved their quality of life dramatically. It is literally the difference between life and death for many.
Some people may feel that the treatment is not for them but everyone should at least have the choice!
Most of all we encourage people with SMA, their friends and family, to work together and fight together for treatment. For the first time ever we have hope! Without everyone getting on board our voices might be lost and the effects of that could be devastating!