What is it about?
Genervon Biopharmaceuticals sponsored a multi-center phase IIA randomized, double-blind placebo controlled clinical trial in which outcomes were compared between 8 ALS patients receiving GM604 and 4 patients receiving placebo for a 2-week period. This trial has demonstrated safety in ALS patients with encouraging signals in slowing the progression of ALS disease by measuring ALS Functional Rating Scale (ALSFRS-R), forced vital capacity, and ALS biomarkers (e.g., TDP-43, tau protein and SOD1).
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Why is it important?
ALS is a devastating life-threatening disease with no cure. ALS is very heterogeneous and involve multiple pathways. The current treatments are all single target treatments working on single pathway. GM604 is a subunit of an endogenous fetal stage motoneuronotrophic factor (MNTF) regulator of the nervous system which works on multiple pathways to treat ALS.
Read the Original
This page is a summary of: 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), F1000Research, March 2017, Faculty of 1000, Ltd.,
DOI: 10.12688/f1000research.10519.1.
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