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This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study (NCT03489200), in which the efficacy of EH301 (1200 mg) was tested in ALS patients

Through its ability to block NOTCH1 maturation, CHAC1/BOTCH was identified as Neuroprotective Gene 7 (NPG7) due to its protective effects in neuronal contexts