The recent decision of the European Medicines Agency (EMA) poses a serious threat to the therapeutic progress achieved by our Duchenne community over the last 25 years.
This decision could jeopardise the small but significant gains made with ataluren. Hundreds of children, adolescents, and adults worldwide are currently being treated with ataluren, some for up to a decade.
We believe it is important to raise our voices to demand that the views of these children, boys and men and their families are taken into account before ataluren is definitively withdrawn.
Join us in a concerted effort to raise awareness and lobby the EMA directly.
Time is of the essence.