Tag

Sod1

All articles tagged with #sod1

health2 years ago•2 min saved

"Promising Daily Treatment Slows Progression of Fatal Neurological Disease"

A new study has shown promise in treating the fatal neurodegenerative disease ALS, by targeting and stabilizing the SOD1 enzyme, which is involved in many cases of the disease. The "molecular stabilizer" S-XL6 successfully restored the protein's function and stopped its toxic effects in mice, rats, and dogs. The researchers are hoping to move the molecule to clinical trials in humans, with the potential to become a co-treatment for Biogen's Qalsody.

via Medical Xpress|

#als #clinical-trials #health

health3 years ago•3 min saved

FDA approves new drug for rare form of ALS, Qalsody.

The FDA has approved Qalsody (tofersen), a new drug to treat a rare form of amyotrophic lateral sclerosis (ALS) caused by the SOD1 mutation, which affects only 2% of the ALS population. The drug has the potential to slow muscle degeneration by targeting SOD1 messenger RNA. Studies showed that the drug reduced plasma neurofilament light (NfL), a blood-based biomarker of axonal injury and neurodegeneration. The drug is given via a lumbar puncture with three doses in 14-day intervals, followed by monthly dosing. Biogen, which makes the drug, will still need to complete additional and larger studies on its effectiveness.

via ABC News|

#als #fda #health