Ionis Pharmaceuticals have announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD).
This is fantastic news for the Alexander disease community and which gives hope to the wider leukodystrophy community.
To read the full press release from Ionis: Ionis AxD press release
There is also a community webinar on Friday 3rd October 2025, at 4pm ET (9pm UK time) to further discuss the results, that can be joined by clicking the following link:
https://ionisph.zoom.us/j/95901687360?pwd=fiKK5nMc4a3YaiGgwBDP8EOyrGHU73.1
