In terms of the high complexity of the purinome comprising diverse nucleotide- and nucleoside-metabolizing enzymes and purinergic receptors differentially distributed on CNS cell types, further intense research has to be performed to elucidate various implications of the purinergic signaling in ALS and evaluate its therapeutic potential in the fight of this relentlessly progressive disease. The gene discussed is P2RX4; the disease is amyotrophic lateral sclerosis.