It was another setback on the family’s quest to save their precious daughter with access to a gene therapy that costs £1.79million per patient – the most expensive drug in the world. It led the family to move from the UK to the US before they secured treatment nearly two years ago. The gene therapy, given as a one-time infusion, works by delivering replacement genes into the ­bloodstream which cause the motor neurons to work normally. But you can stop the damage ­continuing,” said Sally-Anne Tsangarides, of Novartis Gene Therapy that developed the drug. If we could treat a baby at two weeks old with gene therapy they will have suffered much less damage than at nine months.

Source: Daily Mirror May 10, 2021 17:27 UTC