RG3039 is primarily investigated for its potential therapeutic applications in neurodegenerative diseases, particularly those involving motor neuron dysfunction. It has shown promise in preclinical studies for treating conditions like spinal muscular atrophy (SMA) by modulating survival motor neuron (SMN) protein levels, which are critical for motor neuron health. The compound is explored for its ability to enhance SMN2 gene splicing, thereby increasing the production of functional SMN protein. This mechanism could potentially improve motor function and slow disease progression in SMA patients. Additionally, research is ongoing to evaluate its efficacy and safety in other neurodegenerative disorders where motor neuron survival is compromised.