tofersen (Pending FDA Approval)

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Dosing & Uses

Amyotrophic Lateral Sclerosis

Pending FDA approval for superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS)

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Pharmacology

Mechanism of Action

Antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS)

Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.