tofersen

Also known as Qalsody, BIIB067, BIIB-067

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. It is administered as an intrathecal injection.

Key facts

Drug.image: Tofersen.svg
Drug.image_class: skin-invert-image
Drug.width: 300
Drug.tradename: Qalsody
Drug.MedlinePlus: a623024
Drug.DailyMedID: Tofersen
Drug.routes_of_administration: Intrathecal
Drug.ATC_prefix: N07
Drug.ATC_suffix: XX22
Drug.legal_CA: Rx-only
Drug.legal_US: Rx-only
Drug.legal_EU: Rx-only
Drug.CAS_number: 2088232-70-4
Drug.PubChem: 172873401
Drug.DrugBank: DB14782
Drug.UNII: 2NU6F9601K
Drug.KEGG: D11811
Drug.IUPAC_name: all-P-ambo-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'→5')-2'-O-(2-methoxyethyl)-guanylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridine

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Commons category: Tofersen

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Article

6 sections

Contents

Medical uses
History
Society and culture
Economics
Legal status
References

The most common side effects include fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).