Shakil Malji has set up a petition to make Spinraza – the first treatment for SMA – available for people with the condition. His five-month-old daughter, Maryam, has SMA Type 1 – the most severe form of the condition. Without access to the drug, life expectancy for children with SMA Type 1 is rarely longer than two years.
Catherine Woodhead, Chief Executive of Muscular Dystrophy UK, said:
Spinraza offers families hope, but continued delays in making it available are causing heartbreak for families like Maryam’s. It is unthinkable that a child who is now diagnosed with the most severe form of the condition cannot access this treatment in England, yet could if they lived across the border in Scotland. We have seen how effective Spinraza can be in treating people with spinal muscular atrophy, and it is families who suffer when delays happen.
Time is of the essence, and without access to Spinraza babies with the most serious form of the condition are likely to die, while children with other types of spinal muscular atrophy will irreversibly lose the ability to walk, crawl and swallow. Now more than ever, we need to see urgent action from NICE, Biogen and NHS England, who must sit down together to find a solution before any more lives are cut short.
