Ionis have received U.S. FDA Breakthrough Therapy designation for zilganersen for Alexander disease (AxD) - this is the first and only investigational medicine for this condition 💙
To find out more: https://alextlc.org/news/fda-breakthrough-therapy-designation-axd/
#AlexanderDisease #Leukodystrophy

Using #iPSCs from patients with #AlexanderDisease, @umcutrecht.bsky.social scientists discovered that mutations in the protein GFAP disrupt neuroectodermal lineage commitment in neural #organoids, supporting its role as an early modulator of #neurodevelopment.

#neuroskyence #devbio 🧪

Rare Disease : Alexander Disease
For more info, check out NORD and The #AlexanderDisease Lab > alexander-disease.waisman.wisc.edu

#raredisease #chronicillness

We are here to support individuals and families affected by all types of leukodystrophy, get in touch by emailing info@alextlc.org

#alextlc #alexanderdisease #leukodystrophy #helptocope #helptohope