Results showed no significant difference in these scores between those given tofersen or placebo.īut trends toward benefits with tofersen - while not statistically significant - were seen for some secondary measures, including slow vital capacity (a measure of lung function) and hand-held dynamometer (a measure of muscle strength). Its main goal was to assess the effects of 28 weeks of treatment on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) scores, a measure of disability, among people with rapidly progressing disease. Among participants, 60 had rapidly progressing disease, while the remaining 48 had slower-progressing disease. The Phase 3 part enrolled 108 patients, who were randomly assigned to eight spinal canal injections of tofersen (100 mg) or a placebo, given over six months.
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