The FDA has accepted Ionis New Drug Application for zilganersen, the investigational treatment for Alexander Disease, for Priority Review
Community statement from Ionis: alextlc.org/news/ionis-community-sta...
#AlexanderDisease #Leukodystrophy
🔬 How do astrocyte mutations cause brain damage? CADRC REC scholar Dr. Benjamin Clayton is on the case.
With @CWRU & the End Alexander Disease Foundation, his lab is finding ways to restore gene activity and protect white matter in children. #RareDisease #Neuroscience #CADRC #AlexanderDisease
Ionis has opened an Expanded Access Program (EAP) for people with Alexander disease to access zilganersen in the United States
Ionis have released a Community Statement with further information: alextlc.org/news/ionis-zilganersen-p...
#AxD #AlexanderDisease #Leukodystrophy
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: alextlc.org/news/fda-breakthrough-th...
#AlexanderDisease #Leukodystrophy
Figure 3A from the paper linked in the next post: "Prevention and rescue of neural commitment deficits in AxD neural organoids. (A) Protocols that were used to generate neural organoids in the prevention and rescue experiments. For full protocol description, see methods section."
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
