Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Washington University in St. Louis · University of Sheffield · +21 more institutions
Abstract
mutations.
mutations. In each dose cohort (20, 40, 60, or 100 mg), participants were randomly assigned in a 3:1 ratio to receive five doses of tofersen or placebo, administered intrathecally for 12 weeks. The primary outcomes were safety and pharmacokinetics. The secondary outcome was the change from baseline in the cerebrospinal fluid (CSF) SOD1 concentration at day 85. Clinical function and vital capacity were measured.
Citation impact
- FWCI
- 30.49
- Percentile
- 100%
- References
- 27
Authors
31- JMJonathan MillCorresponding
Washington University in St. Louis, University of Sheffield
- MCMerit Cudkowicz
Harvard University, Massachusetts General Hospital, University of Sheffield
- PJPamela J. Shaw
Sheffield Teaching Hospitals NHS Foundation Trust, University of Sheffield
- PMPeter M. Andersen
Umeå University, University of Sheffield
- NANazem Atassi
Harvard University, Massachusetts General Hospital, University of Sheffield
Topics & keywords
- Medicine
- Placebo
- SOD1
- Amyotrophic lateral sclerosis
- Adverse effect
- Randomization
- Internal medicine
- Confidence interval
- Good health and well-being