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Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
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Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 07 09; 383(2):109-119.
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PubMed
subject areas
Adult
Amyotrophic Lateral Sclerosis
Disease Progression
Dose-Response Relationship, Drug
Double-Blind Method
Female
Headache
Humans
Injections, Spinal
Intermediate Filaments
Leukocytosis
Male
Middle Aged
Mutation
Oligonucleotides
Oligonucleotides, Antisense
Vital Capacity
authors with profiles
Merit Ester Cudkowicz, M.D.