The BDFA is delighted to announce that in the last week we have received very positive news from the European Medicines Agency (EMA) and the U.S Food and Drug Administration (FDA) on the approval of Brineura as the first treatment for children with CLN2 (Late Infantile Batten disease).
The results from a clinical trial in Europe and the USA has shown Brineura to slow down the rapid, devastating progression of the disease and gives hope to many families where previously there was none.
Over the last four years the BDFA has worked very closely with the pharmaceutical company, BioMarin, clinicians, and our colleagues in the Batten disease patient organisation community to deliver this day but we have much more work to do to ensure that the treatment is available to those children and families who need it. Our heartfelt thanks also go to those families who have taken part in the clinical trial and their commitment to not only their own children but all children living with this disease.
More details on these announcements can be found at: