A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604 in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001) and a single compassionate patient treatment (Protocol GALS-C).

F1000Res · 2017 · PMC6051227 · PMID 30057745

Europe PMC DOI

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highly significantno p-value reported

Biomarker: Biomarkers are not the gold standard but highly significant to proof not only MOA but modulation of critical corrupted disease specific gene/protein expression of treated ALS patients (not in vitro or in vivo studies).

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