RG7800 is primarily investigated for its potential therapeutic applications in treating neurodegenerative diseases, particularly spinal muscular atrophy (SMA). It functions as a small molecule modulator of the splicing of the SMN2 gene, which is crucial for increasing the production of survival motor neuron (SMN) protein. This mechanism is vital because SMA is characterized by a deficiency in SMN protein, leading to motor neuron degeneration and muscle weakness. By enhancing SMN protein levels, RG7800 aims to improve motor function and slow disease progression in SMA patients. Its development represents a promising approach in the field of genetic medicine, offering hope for effective treatment options for this debilitating condition.