A toddler unable to move because of a deadly muscular condition has made amazing strides after a $2million infusion of the world’s most expensive drug.
Maddy Smith, from Blaine, Minnesota, was unable to crawl on her second birthday, and could not speak or eat solid food.
But just six months later she is walking around with a huge smile on her face and laughing.
Her incredible recovery from Spinal Muscular Atrophy (SMA) has been credited to a one time infusion of a $2million gene therapy treatment called Zolgensma.
Maddy was given the drug on the eve of her second birthday – just one day before the cut-off point beyond which Zolgensma cannot be used.
And her parents have credited the drug with transforming their daughter’s life.
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‘I’m ecstatic. I’m so happy and thankful…
‘There were a lot of sleepless nights fighting for her. Fighting for this
‘People everywhere are celebrating with us and everyone who fought for her to get Zolgensma, this is this is what it does.
‘This is what they gave her.’
Left untreated, SMA stops sufferers walking, holding their heads up, clearing their airways or swallowing.
It can be deadly in the most serious cases.
Maddy is one of the first children in the world to be treated with Zolgensma, which was paid for by her family’s state insurance after a legal battle.
Doctors are unsure what her long term prognosis is – but are now hopeful Maddy could make the same progress as a healthy child her age.
The toddler’s physician Dr Peter Karachunski from the University of Minnesota Masonic Children’s Hospital said: ‘It’s just pure happiness.
‘To see someone with improvement, such dramatic improvement, essential normal milestones is certainly very impressive.’