NHS approved to use gene therapy to treat baby born with spinal muscular atrophy
The parents of a baby with a fatal condition have succeeded in their campaign for their son to be treated with the world’s most expensive drug.
A new gene therapy, Zolgensma, will be used to treat 10-month-old Edward, from Colchester, who has severe spinal muscular atrophy (SMA), after his parents were given the green light earlier this week. The genetic condition, which is caused by a missing protein, weakens the muscles and affects movement and breathing.
