As an example, in SMA drug discovery, the 3,6-disubstituted pyridazine was identified through High-throughput screening in the NSC-34 cell line containing a SMN2 minigene reporter, and was then chemically modified leading to the synthesis of Branaplam (nowadays in clinical trial phase 2 for SMA treatment) (see section Direct and Indirect Modulation of Survival Motor Neuron 2 Transcription) (Cheung et al., 2018). Here, SMN2 is linked to proximal spinal muscular atrophy.