Yesterday, Wednesday 13 Nov 2019, the first infusion of Brineura was given to a child at Great Ormond Street Hospital as part of the new Managed Access Agreement between BioMarin and NHS England. The child would not have been able to receive the treatment without this Agreement in place.
This is a major milestone in the treatment of CLN2 Batten disease and we are grateful to everyone who worked so hard to reach this point.
We also think of those children for whom this day has come too late and send their families our best wishes.
There will be more news about the progress of Brineura in the near future so watch this space.