ABSTRACT Risdiplam is a new therapeutic agent developed to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease caused by mutations in the SMN1 gene. Unlike previous invasive therapies, risdiplam offers the advantage of oral administration, significantly improving patient comfort and accessibility. The review provides information on an SMA historical overview, breakthrough therapies of the development, and design of the methods used to treat SMA. We then focus on its structure and physicochemical properties. The analysis of risdiplam concentrates on developing improved analytical methods for the precise quantification of risdiplam and its metabolites by high‐performance liquid chromatography with mass spectrometry in biological samples using octadecyl stationary phases. For sample preparation, only the protein precipitation method was used. Challenges associated with the risdiplam analytics include developing a highly sensitive and selective method in biological matrices and dealing with potential interferences from the biological matrix. Future research should focus on improving analytical methods, investigating metabolite activity, and expanding our knowledge of its long‐term effects.