just approved Qalsody (tofersen) for with mutation (~2% of ALS cases), based on a ⬇️ of the neurofilament (NFL) in the plasma.
🔹 ALS is a progressive neurodegenerative disorder, attacking nerve cells that control voluntary muscles (e.g., breathing)
🔹 Qalsody is given intrathecally (in the spine) with 3 initial doses at 14-day intervals and maintenance every 28 days
🔹 Most common side effects were pain, fatigue, joint pain, ⬆️ white blood cells in the CSF, and muscle pain

#FDA #als #sod1 #Biomarker

Last updated 2 years ago

The EMBO Journal · @embojournal
383 followers · 70 posts · Server sciencemastodon.com

Cover feature in issue 42/2:

sensor controlling ‘mucin
by Tal Ilani, Deborah Fass and colleagues with SciStories.com

And:
Gut colonization via -less EF-G paralog
cells in two minds about start
/#PP2A module restraining -Akt
Zinc-dependent structural transition in ALS-mutated
embopress.org/toc/14602075/202

#golgi #oxygen #intestinal #glycosylation #gtpase #yeast #cellcycle #mastl #pi3k #sod1

Last updated 2 years ago