By confirming the dual, early-beneficial and late-detrimental role possibly played by P2X7 in ALS, we have recently demonstrated that the direct activation of the receptor by the agonist 2′(3′)-O-(4-benzoylbenzoyl) adenosine 5′-triphosphate during the pre-symptomatic phase is ameliorative by inducing the improvement of the innervation and metabolism of the myofibers, the proliferation/differentiation of the satellite cells, and the prevention of the denervation atrophy of skeletal muscles in SOD1-G93A mice [20]. The gene discussed is P2RX7; the disease is amyotrophic lateral sclerosis.