Brineura, already given to children with life-limiting genetic disease, to be injected into back of eye in Great Ormond Street trial
Eight children born with a “devastating” genetic disease in England have become the first in the world to receive a “pioneering” treatment aimed at stopping them from going blind.
Doctors at Great Ormond Street hospital in London are trialling a drug they believe may save the eyesight of the children who have CLN2-type Batten disease. Brineura, already successful in animals, is being administered to four boys and four girls on a compassionate use basis.
