FDA approves new drug for rare form of ALS, Qalsody.
TL;DR Summary
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.
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