Zolgensma is a revolutionary gene therapy that can stop a deadly childhood condition called SMA in its tracks. It’s also one of the most expensive drugs in the world
Elizabeth Wraige remembers the first time she delivered the diagnosis. “Parents feel as if they’ve been hit by a sledgehammer,” she says. Her patient, a baby boy, had been born tiny and perfect to overjoyed parents six months earlier. But they had begun to feel something was not right. He seemed floppy and was not moving normally. Tests showed he had spinal muscular atrophy (SMA), the most deadly genetic condition in children under two, in which a deficit of a crucial protein causes motor neurons to die, and the body slowly loses the ability to move. Babies with untreated type 1 SMA, the most severe form, will never sit, crawl or speak and are slowly robbed of the ability to move, swallow and breathe. Most die before they are two.
“In that conversation, any hope that it was something that could be remedied was taken away,” says Wraige, a paediatric neurologist at Evelina London children’s hospital. “It was devastation.” She could offer only one shred of consolation: SMA, an incurable condition that affects one in 6,000-10,000 children, is at least not painful.
Arnaud Robert and Paolo Woods write: Zach, who lives in the suburbs of Chicago, was born with SMA, and given Zolgensma in October 2019, when he was eight months old. His father’s trucking union funded the millions needed. Zach also takes a daily medication.
