When it comes to modeling the pathophysiology and potential treatments for SMA, IGHMBP2-related disorders, and other neuromuscular disorders, in vitro modeling systems allow for efficient and high-throughput screening of therapeutics in the context of real patients by utilizing patient-derived cells featuring a variety of disease-causing mutations [19,20,21]. Here, IGHMBP2 is linked to proximal spinal muscular atrophy.