Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis

Neurological Sciences, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 17, 2025

Abstract Objective Tofersen, an antisense oligonucleotide, has recently received FDA and EMA approval for treating amyotrophic lateral sclerosis (ALS) in adults with SOD1 gene mutations. This systematic review meta-analysis synthesized evidence on tofersen’s safety efficacy patients -related ALS. Methods A comprehensive search of three databases was conducted from inception through October 2024. Eligible studies included clinical trials, observational studies, case studies. Meta-analyses were using a random-effects model RevMan. Results Twelve involving 195 treated tofersen met the inclusion criteria, comprising two randomized controlled trials (RCTs), five cohort one series, four reports. Tofersen demonstrated promising effects, notably reducing levels cerebrospinal fluid neurofilament light chain (NfL) plasma, biomarker strongly correlated ALS progression survival. Meta-analysis RCTs showed significantly lower rate decline Functional Rating Scale-Revised (ALSFRS-R) scores baseline group compared to placebo (SMD = 0.44, 95% CI [0.05 0.83], P 0.03) significant reduction predicted Slow Vital Capacity ( 0.005). In pre-post decrease (ALSFRS-R rate) observed (MD -0.28, [-0.40 -0.15], < 0.0001). Reported adverse events consistent or procedural effects. Conclusion Current suggests that effectively reduces NfL slow disease ALS, showing promise as targeted therapeutic option.

Language: Английский

Effects of virtual reality mindfulness on cognition and well-being in ALS: A randomized trial protocol

Contemporary Clinical Trials, Journal Year: 2025, Volume and Issue: 152, P. 107876 - 107876

Published: March 5, 2025

Language: Английский

Effects of Respiratory Training on Pulmonary Function, Cough, and Functional Independence in Patients with Amyotrophic Lateral Sclerosis

Neurology International, Journal Year: 2024, Volume and Issue: 16(6), P. 1332 - 1342

Published: Nov. 1, 2024

Respiratory complications in patients with amyotrophic lateral sclerosis (ALS), due to the involvement of respiratory muscles, are leading cause death, and physiotherapy (RP) focuses on addressing these complications.

Language: Английский