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#FDA just approved Qalsody (tofersen) for #ALS with #SOD1 mutation (~2% of ALS cases), based on a of the #biomarker 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